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I’m Putting My Faith in Science After My Son’s Diagnosis
I can’t put into words how gut-wrenching it was to receive my son’s sickle cell ...
More from Sickle Cell Disease News
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US Patient Groups Give Thumbs-Up to Rule Against Surprise Billing
The National Organization for Rare Disorders (NORD) is applauding the Biden administration for announcing a ... -
Sickle Cell Disease Won’t Stop Me From Succeeding
Last week, I did something I never thought I would do. It’s something that people ... -
UCSF Awarded Nearly $8.4M for CRISPR_SCD001 Gene Editing Trial
The University of California, San Francisco (UCSF) has been awarded $8.39 million to fund a ... -
Summer Is Here, So No More Sickle Cell Crises, Right?
It’s summertime, my favorite season here in the U.K. Summer brings great music, good vibes, ... -
My Concerns as COVID-19 Restrictions Are Lifted in the UK
Most COVID-19 restriction laws were lifted in England on July 19. This includes mask requirements, ... -
Partnership Aims to Lower Out-of-Pocket Costs for Rare Disease Meds
AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology ... -
To Me, Vegan Diets Aren’t Ideal for People With Sickle Cell Disease
I can barely watch five minutes of television before an advertisement for a new vegan ... -
Please Be Mindful When Speaking to Someone With a Chronic Illness
When speaking to a person with a chronic illness, you might do and say things ... -
Emmaus Seeks Approval of Oral Medication Endari in Kuwait
Emmaus Life Sciences has submitted an application seeking the approval of its oral medication Endari ... -
I’m Cautiously Optimistic as COVID-19 Restrictions Ease in the UK
COVID-19 restrictions are easing here in the U.K. Prime Minister Boris Johnson said we must ... -
Nominations Open for 2022 Eurordis Black Pearl Awards
Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. ... -
‘Cheek Week’ Seeks Donors to Join Marrow Donor Program
As a stem cell transplant is the only curative treatment for sickle cell disease (SCD), ... -
Does Everyone With Sickle Cell Need a Folic Acid Supplement?
It’s one thing to deal with the emotional and psychological trauma that comes with a ... -
Study to Evaluate Using JSP191 With ARU-1801 as Gene Therapy
A new research collaboration between Jasper Therapeutics and Aruvant Sciences will evaluate the use of ... -
Growing Up With Sickle Cell Taught Me to Stand Up to Adversity
With a chronic condition like sickle cell disease, I sometimes find it difficult to look ... -
Why I’m Open About My Experiences With Sickle Cell
One of the many stereotypes of British people is that we can be extremely reserved. ... -
CTX001 Continues to Show Promise in Severe SCD
A single dose of CTX001, an experimental gene-editing cell therapy, rapidly increases the levels of ... -
Artist Has Vision to Open People’s Minds to Sickle Cell Disease
A Connecticut-based artist living with sickle cell disease and blindness in his right eye is ... -
Mother Creates Oklahoma Sickle Cell Nonprofit to Give Families Hope
Velvet Brown-Watts was devastated when her son, Jeremiah Watts Jr., now 16, was diagnosed with ... -
Centogene Sets Mission to Cure 100 Rare Diseases in Next 10 Years
At its first virtual investor event, biotech company Centogene set a bold mission: to cure ... -
How I Strengthen My Son’s Immune System to Keep Him Crisis-free
The trouble with sickle cell disease is that it’s challenging to determine which end of ... -
The Ways I Made Hydration a Habit
I can’t stress enough the importance of drinking water. Experts recommend that normal, healthy adults ... -
9 Nonprofits Working in SCD Care Awarded GBT ACCEL Grants
To improve life for people with sickle cell disease (SCD), Global Blood Therapeutics (GBT) has awarded ... -
How My Origin Story and Sickle Cell Disease Shaped My Childhood
I am going to take you behind the scenes of how this column, “Sickle Sagas,” ... -
Welcome to ‘I Sickle Well,’ a Chronicle of a Mother’s Caregiving Journey
Would I be a good mother to him? Would he have a good quality of ... -
Improving Care for ‘Patients of All Ages’ Is GBT’s Goal, Exec Says
Global Blood Therapeutics (GBT) is currently focused on expanding the use of Oxbryta (voxelotor) to children, ages 4–11, ... -
EveryLife Introduces First of Kind ‘Roadmap’ to ICD Codes
To help patient advocacy leaders and their partners better understand how global health statistics codes ... -
Bluebird to Resume LentiGlobin Trials After FDA Lifts Hold
Bluebird Bio is set to resume trials of LentiGlobin, its investigational gene therapy for sickle cell ... -
Do We Have to Die for Change?
June 19 was both World Sickle Cell Day and Juneteenth! It wasn’t until last year ... -
For World Sickle Cell Day, Read About My Reality
World Sickle Cell Day is upon us tomorrow, June 19. Following are memories that illustrate ... -
Oxbryta in HOPE-KIDS Meeting ‘Urgent Need’ of Children, Ages 4-11
In children with sickle cell disease (SCD), ages 4–11, weight-based doses of Oxbryta (voxelotor) result ... -
Get Ready for NORD’s Patient and Family Forum, Set for June 26-27
Those who wish to gain practical tools for living optimally with rare diseases are encouraged ... -
Oxbryta Given Promising Innovative Medicine Status in UK
A Promising Innovative Medicine (PIM) designation has been granted to Oxbryta (voxelotor), a treatment to ... -
Welcome to ‘Sickle Sagas’: Documenting Life With My Disorder
Recently I have been engrossed in sagas about the viking king Ragnar Lothbrok as well ... -
Rare 2030 Action Campaign Pushing for European Plan for Rare Diseases
Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European ... -
Sickle Cell Disease Is an Inseparable Part of My Identity
Many people with chronic illnesses may agree with a common statement that “our illnesses do ... -
Supporters Urged to Wear Red for World Sickle Cell Day on June 19
From sporting red gear to entering a spoken word contest, supporters globally are poised to ... -
How to Prepare Ahead of Time for Sudden Hospitalizations
One of the most annoying things about sickle cell disease is that a hospitalization can ... -
Advocates Can Try for Grants Ahead of Rare Disease Week on Capitol Hill
Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the ... -
Machine Learning Can Predict Rapid Kidney Function Decline
Machine learning tools can identify sickle cell disease (SCD) patients at high risk of progressive ... -
Let’s Respect That ‘Healthy’ Means Something Different to Everyone
There is no single weight, shape, size, age, or ability that dictates healthiness — no ... -
Endari Sickle Cell Therapy Now Under Review in Saudi Arabia
The Saudi Food and Drug Authority (SFDA), in Saudi Arabia, will review Emmaus Life Sciences’ ... -
Imaging Technique Predicts Risk of Vision Problems
Rapid and repeated imaging of blood flow through the eyes can inform physicians of a ... -
Why We Need Better Doctor-patient Communication
It has been a while since I last wrote. This is because unfortunately, I had ... -
Gene Therapy ARU-1801 Showing Early Efficacy, Safety in Phase 1/2 Trial
ARU-1801, an experimental gene therapy for sickle cell disease (SCD), lowered the number of vaso-occlusive crises ... -
When Health Crises Strike, We Must Focus on Healing
Unfortunately, the healing process is not always as straightforward as we’d like it to be. ... -
US Nonprofits Team Up to Bolster Advocacy Efforts
Two U.S. nonprofits are partnering to tackle legislative issues that benefit sickle cell disease (SCD) ... -
Oxbryta Safe and Effective in Children Ages 4 to 11, Early Trial Data Indicates
Six months of treatment with Oxbryta (voxelotor) safely and effectively increased hemoglobin levels and reduced ... -
I Struggled to Take My Meds — Until It Affected My Pocketbook
In 2005, my doctors started me on a medication called hydroxycarbamide, also known as hydroxyurea, ... -
Non-viral Gene Editing of Marrow Stem Cells Shows Potential in Early Study
A non-viral, CRISPR-based gene-editing tool showed a potential to reverse mutations that cause sickle cell ... -
Spoken Word Poetry Contest Open to Sickle Cell Patients, Caregivers
A spoken word poetry contest is welcoming submissions from people with sickle cell disease (SCD) ... -
Should You Blog About Your Health Journey?
Recently, a fellow sickle cell warrior messaged me on Instagram to ask if I thought ... -
FDA Expands Approvals of Ferriprox
The U.S. Food and Drug Administration (FDA) has expanded the label of Chiesi Global Rare ... -
FDA Clears Way for Phase 1 Trial of Low-dose Oral Carbon Monoxide
The U.S. Food and Drug Administration has cleared the way for a Phase 1 trial ... -
Finding Sickle Cell Awareness in Unexpected Places
I have a lot of pride when it comes to my general knowledge. I think ... -
Mitochondrial DNA Carried in Blood May Trigger Inflammation in Sickle Cell
People with sickle cell anemia have higher levels of mitochondrial DNA (mtDNA) — DNA specific to ... -
Quick Approval Given COVID Vaccines Raise Concerns, Rare Disease Patients Say
More than half of the people with rare diseases and their caregivers, asked in a ... -
Cancer Diagnosis in Patient Given LentiGlobin Revised to Severe Anemia
A patient diagnosed initially with the blood cancer myelodysplastic syndrome (MDS) after receiving LentiGlobin, Bluebird ... -
I’m Having Trouble Accepting a Recent Avascular Necrosis Diagnosis
When I go to my doctors with a problem, I’m never under the impression that ... -
‘Real Impact’ Grants Help Nonprofits Help People With Blood Disorders
Imara has opened applications for its second-annual “Real Impact” grants for community-based nonprofit organizations that ... -
EveryLife Scholarship Fund Open Until May 7 for Adults With Rare Diseases in US
The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to ... -
Partners Seek to Educate About Pediatric Blood Transfusions
To inform sickle cell disease (SCD) patients and their caregivers about blood transfusions, which are commonly ... -
What Playing Football Made Me Realize About Myself
For the first time in my life, I played a game of football, or soccer ... -
Oxbryta Increased Hemoglobin, Reduced Hemolysis in Adults and Adolescents
Treatment with oral Oxbryta (voxelotor) leads to rapid and sustained rises in hemoglobin levels, reduces red ... -
How to Cultivate a Successful Career While Living With Sickle Cell Disease
I want to start by stressing that I do not believe a booming career is ... -
FT-4202 at Higher Dose Safe and Helpful to Patients in Phase 1 Trial
A higher 600 mg daily dose of FT-4202, a potential disease-modifying oral treatment for sickle ... -
Getting Vaccinated Was a Major Mental Hurdle to Clear
Last Wednesday, I received my second and final dose of the COVID-19 vaccine. Because I ... -
GBT Granted Rights to SCD Small Molecule Programs in Deal With Sanofi
Global Blood Therapeutics (GBT) has been granted exclusive global rights to two small-molecule research programs ... -
1st CRISPR Gene Editing Trial Slated to Open in Severe SCD Patients
The U.S. Food and Drug Administration approved the start of a first clinical trial of ... -
Cost of Rare Diseases in US? Nearly $1 Trillion in 2019, EveryLife Foundation Finds
As anyone affected by a rare disease knows, treating the illness while trying to go ... -
My Views About Exercise Changed as I Grew Older
Sickle cell disease and exercise were antonyms for me growing up. I was raised with ... -
Imara Opens Higher Dose Arms in Phase 2b Trials of IMR-687
Imara is opening higher dose arms in two Phase 2b trials assessing the safety and ... -
Graphite Bio Raises $150M to Advance GPH101, Other Gene-editing Therapies
Graphite Bio has raised $150 million in Series B funding to expand and advance the ... -
What I’ve Learned a Year After the Pandemic Started
I started to shelter at home due to the pandemic in mid-March 2020, and I’m still ... -
Veto T-cells Boost Success of Stem Cell Transplant for SCD
Adding Cell Source’s special class of immune cell, Veto T-cells, to blood stem cells from an ... -
What a Top-up Blood Transfusion Looks Like for Me
I am scheduled to have a blood transfusion today. So, I want to take this ... -
GBT Welcomes Proposals for ACCEL Grants, Aiding Sickle Cell Community
Global Blood Therapeutics (GBT) has opened its grant program — the Access to Excellent Care ... -
I’ve Come to Realize That We’re All in This Fight Together
While sickle cell disease is considered a rare disease, and is recognized each year at ... -
Registration Open for 8th Annual ‘Walk With The Stars’ Event
Registration is open for the 8th annual “Walk with the Stars,” one of the largest ... -
Gene Therapy LentiGlobin Unlikely Cause of Blood Cancer in Early Trial, Bluebird Says
LentiGlobin, Bluebird Bio’s investigational gene therapy for sickle cell disease (SCD), is unlikely to be ... -
Pink, Green, Blue and Purple Lights Illuminated World for Rare Disease Day
Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is ... -
Symposium Explores Social Burden Surrounding Access to Pain Meds
The opioid crisis, along with the stigma of suspected drug use, and racial bias all ... -
It’s Difficult to Manage Sickle Cell Pain at Home During a Pandemic
I have not felt well recently. I have been struggling with a lot of pain ... -
VEST Lowers Duration of Painful SCD Crises
Vascular electrical stimulation therapy (VEST), a form of local electrical stimulation applied to the skin, ... -
Why It’s Important for Everyone to Prioritize Their Health
It’s easier said than done. Everyone always says that health should be a person’s No. ... -
Stem Cell Transplant, Gene Therapy in SCD Tied to Improved Quality of Life
The quality of life of people with sickle cell disease (SCD) and transfusion-dependent thalassemia (TDT) ... -
Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion
Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of ... -
Rare Disease Day at NIH, Set for March 1, Growing Year by Year
Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place ... -
Gates Foundation, Novartis Working on Affordable Gene Therapy for Sickle Cell
Novartis and the Bill and Melinda Gates Foundation are collaborating to develop a single-shot gene ... -
Here’s How You Can Be a Better Partner for a Sickle Cell Patient
In my last column, I shared tips about dating with sickle cell disease. Continuing in ... -
Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients
People with rare disorders have a worse healthcare experience than those affected by chronic diseases, ... -
Bluebird Bio Pauses Trials of Potential Gene Therapy LentiGlobin
Bluebird Bio has paused two clinical trials of LentiGlobin, its gene therapy candidate for sickle ... -
Our Wellness Depends on Our Ability to Practice Self-love
Self-love is so important. I read somewhere that we teach others how to love us ... -
Alternatives to Opioids Seen to Ease Pain, Raise Serotonin Levels in Early Study
A good diet and companionship, or treatment with an antidepressant called duloxetine was seen to ... -
Oxbryta Can Ease or Fully Heal Leg Ulcers, Trial Analysis Shows
Oxbryta (voxelotor) can help ease, or fully heal, painful leg ulcers in people with sickle ... -
It’s OK to Take a Break From Raising Awareness About Sickle Cell Disease
I am passionate about raising sickle cell disease awareness. Increased awareness leads to a better ... -
Pandemic Won’t Stop Rare Disease Day on Feb. 28
Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 ... -
NHLBI Challenges Students to Create Innovative Tools to Raise Awareness
A new National Heart, Lung, and Blood Institute (NHLBI) challenge will award a first prize ... -
NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns
While progress was made last year on newborn screening and other policy issues critical to ... -
Here’s What I’ve Learned About Dating With Sickle Cell Disease
One thing people always ask me is how I navigate dating as a sickle cell ... -
Abnormal Flow of Sickled Cells Damages Blood Vessel Walls
Red blood cells in people with sickle cell disease (SCD) flow through blood vessels in ... -
Even During Sickle Cell Crises, I Avoid the Hospital Due to the Pandemic
This week has been challenging for me. A brutal combination of cold weather and stress has ... -
Lonza to Produce Aruvant’s ARU-1801 for Pivotal Trial
A recent manufacturing agreement between Aruvant Sciences and Lonza will advance the development of ARU-1801, ... -
EMA Reviews Oxbryta: Therapy Would Be 1st in EU Targeting SCD Root Cause
The European Medicines Agency (EMA) is reviewing an application from Global Blood Therapeutics (GBT) that ... -
Trial Will Test New Combination Regimen for Stem Cell Collection
A clinical trial will assess a combination of MGTA-145 plus plerixafor as a new regimen ... -
Sickle Cell May Knock Me Down, but I Will Stand Back Up
In a previous column, I mentioned some of the good things that happened to me ... -
FDA Clears Way for Trial of Gene Editing Therapy for Severe SCD
The U.S. Food and Drug Administration (FDA) has cleared the initiation of a Phase 1/2 ... -
I Can Still Be Productive During a Sickle Cell Crisis
I’m writing this column during a sickle cell disease crisis. I have not had one ... -
Clinical Trial Cleared for GPH101, First Potentially Curative SCD Therapy
The investigational gene editing therapy GPH101 will be the first potentially curative treatment for sickle ... -
BCH-BB694 Gene Therapy Safely Treating Severe SCD Patients in Pilot Trial
BCH-BB694 — an experimental gene therapy targeting the BCL11A gene — safely increased the levels ... -
Oral IMR-687 Seen to Lessen VOCs, Pain Crises in Patients in Phase 2a Trial
Six months of treatment with oral IMR-687 safely and effectively reduced the number of vaso-occlusive crises ... -
2020 Was Challenging, but I’m Grateful for My Progress
Just like that, 2020 is over. What a year! I do not think 2020 turned ... -
Oral FT-4202 Safe, Found to Improve Red Blood Cell Function in SCD
FT-4202, a potential disease-modifying oral treatment for sickle cell disease (SCD), is well-tolerated in people with ... -
Top 10 Sickle Cell Disease Stories of 2020
Sickle Cell Disease News brought you daily coverage of key findings, treatment developments, clinical trials, ... -
US Army Begins Testing Recruits for Sickle Cell Trait
To identify at-risk service members, the U.S. Army has begun testing recruits for the sickle ... -
Tips for Staying Healthy This Holiday Season
Here’s a little fun fact about me: Christmas is my second favorite time of year ... -
CTX001 Continues to Prevent VOCs in People With Severe Disease, Trial Finding
CTX001, an experimental gene-editing cell therapy, safely and effectively increased the levels of fetal hemoglobin ... -
Emmaus Support Program Gives US Patients Low-cost Access to Endari
Emmaus Life Sciences has launched a program that will allow eligible sickle cell disease patients ... -
Oxbryta May Be SCD Blood Transfusions Option in Pandemic, Case Report Finds
Treatment with Oxbryta (voxelotor) may be a feasible alternative to blood transfusions for sickle cell ... -
SCD Patients Benefit from Early Rivipansel Treatment for VOCs, New Analyses Show
Starting treatment with rivipansel (GMI-1070) shortly after vaso-occlusive crisis (VOC) onset significantly shortened hospital stays ... -
GBT Launches Early Access Program for Voxelotor in Europe, Other Regions
Global Blood Therapeutics (GBT) has launched an early access program for voxelotor in Europe and ... -
The Lessons I’ve Learned as a Columnist
I have been writing for BioNews, the publisher of Sickle Cell Disease News, for over ... -
Hemex Scientists Awarded $100K for Noninvasive Diagnostics Tool for SCD, Other Disorders
Hemex Health was awarded third place and $100,000 in a National Institutes of Health (NIH) ... -
Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe
The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and ... -
We Go Through Life at Our Own Pace
I hate when sickle cell disease gets in the way of life, although I feel ... -
Gates Foundation Funds Studies Into CRISPR/Cas9 Gene Editing for SCD
Intellia Therapeutics has received a grant from the Bill & Melinda Gates Foundation to develop ... -
Switching Treatments Helped to Reduce My Iron Overload
Iron overload is a side effect that some sickle cell patients suffer from as a ... -
ARU-1801 Gene Therapy Granted Orphan Drug Status in EU
The European Medicines Agency (EMA) has granted the designation of orphan drug to ARU-1801, an ... -
Why I Will Be Getting the COVID-19 Vaccine
On Nov. 9, Pfizer and BioNTech announced that their COVID-19 vaccine was found to be more ... -
Rifaximin, Oral Antibiotic, Named Orphan Drug for Potential to Ease Pain Crises
The U.S. Food and Drug Administration (FDA) has designated rifaximin, an oral antibiotic, an orphan drug as a ... -
FT-4202, Oral SCD Therapy in Clinical Testing, Named Orphan Drug in EU
FT-4202, a potential disease-modifying oral treatment for sickle cell disease (SCD) by Forma Therapeutics, has been ... -
‘Disabled’ Is Not a Bad Word
“Disabled” is not a pejorative, but it definitely felt like one when I was growing ... -
NIH Funds New Project to Improve SCD Care for Children in Malawi
A team of researchers led by UNC Project-Malawi has been given a one-year, $500,000 grant ... -
What Does It Mean to Be Successful?
Recently, I was playing a question-and-answer game with my partner, and one question that came ... -
CSL889 Designated as Orphan Drug in Europe and US for Treating Sickle Cell Disease
The European Commission and the U.S. Food and Drug Administration have granted orphan drug status ... -
Preparing for Lockdown 2.0
COVID-19 cases are increasing again here in the U.K., and government officials can no longer ... -
EU Approves Adakveo to Treat Pain Crises in SCD Patients, 16 and Older
The European Commission has approved Novartis’ Adakveo (crizanlizumab) as a preventive treatment for recurrent vaso-occlusive ... -
When Feeling Overwhelmed, Here’s How I Reduce Stress
I have felt pretty overwhelmed lately. After months of sheltering at home and being relatively ... -
Phase 1 Trial of Oral FTX-6058 to Promote Fetal Hemoglobin Set to Open
FTX-6058, an investigational oral treatment being developed by Fulcrum Therapeutics for sickle cell disease (SCD) and beta-thalassemia, is entering ... -
How I Manage a Sickle Cell Crisis at Home
In a previous column, I spoke about some of the things that trigger a sickle ... -
Siklos, Hydroxyurea-based Treatment for Pediatric SCD, Available in 100 mg Scored Tablets
Siklos (hydroxyurea) — indicated for the treatment of children 2 and older with sickle cell ... -
While My Health Challenges Are Tough, I Smile Through Life
One of my favorite features is my smile, and I find that in life, I ... -
Cyclerion Suspends Development of Olinciguat for Sickle Cell Disease
Cyclerion Therapeutics has decided to suspend the development of olinciguat, its investigational oral therapy for ... -
$7.1M NIH Grant Supports Study of Opioid Alternatives to Manage SCD Pain
The National Institutes of Health (NIH) has awarded a $7.1 million grant to University of Illinois Chicago (UIC) researchers ... -
Why I Share My Health Journey on Social Media
I share much about my journey with sickle cell disease on social media. I find ... -
C-Path Launches Consortium to Support Sickle Cell Therapy Development
A new consortium has been launched by the Critical Path Institute (C-Path) to support the ... -
Here’s What Triggers a Sickle Cell Crisis for Me
A sickle cell crisis is one of the main symptoms sickle cell patients face. However, ... -
Rivipansel Granted FDA’s Rare Pediatric Disease Designation for SCD
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to GlycoMimetics’ investigational ... -
How to Be a Good Friend to Someone With Sickle Cell
Friendships are beautiful and make life much more meaningful. I love being in the company ... -
Fulcrum’s FTX-6058 Shows Promising Efficacy as SCD Therapy in Disease Models
FTX-6058, an investigational treatment for sickle cell disease (SCD) and beta-thalassemia being developed by Fulcrum ... -
Medications Activating sGC Protein May Have Promise as SCD Therapy, Study Shows
Medications that activate the protein soluble guanylyl cyclase (sGC) may have promise in the treatment ... -
Saying I’m Fine When I’m Not to Avoid the Hospital
Needing to go to the hospital due to a vaso-occlusive crisis is never ideal or ... -
Gene Therapy LentiGlobin Granted Priority Medicines Designation for SCD in EU
Bluebird Bio’s investigational gene therapy LentiGlobin has been granted priority medicines (PRIME) designation by the European Medicines Agency (EMA) for ... -
Gene-editing Therapy CTX001 Granted Priority Medicines Designation for Severe SCD in EU
CTX001, an investigational gene-editing cell therapy, has been granted priority medicines (PRIME) designation by the ... -
I Started Going Outdoors More, but Soon Changed My Mind
After many months of sheltering in place, or shielding, as we call it here in ... -
Graphite Bio Raises $45M to Move New Way of Gene Editing Into Sickle Cell Trial
Graphite Bio, a startup biotech company, has raised $45 million in an initial financing round ... -
Trying a Different Medication to Address Iron Overload
Iron overload is a common side effect for sickle cell patients participating in blood transfusion ... -
GreenLight Wins $3.3M Grant to Pursue mRNA-based Gene Therapies
The Bill & Melinda Gates Foundation has awarded a $3.3 million grant to GreenLight Biosciences ... -
Thinking Ahead by Cultivating Healthy Habits Now
Having a lifelong condition like sickle cell disease forces people to grow up pretty quickly. ... -
Study Identifies FDA-approved Medications With Potential for Treating SCD
Using a newly developed assay, researchers identified seven U.S. Food and Drug Administration (FDA)-approved medications ... -
Activities Underway for Newborn Screening Awareness Month
As it does each September during Newborn Screening Awareness Month, Baby’s First Test is sharing information ... -
Genetic Differences Seen to Mirror Sickle Cell Severity in African Study
Genetic analyses of African patients identified several genes and biological pathways that are associated with ... -
My 2 Hopes for Sickle Cell Awareness Month
Just like that, it’s September again – aka Sickle Cell Awareness Month. Sickle cell awareness ... -
‘Brain-Gut Axis’ May Be What Links Stress and Crises in SCD Patients
Microbes in the gut play a key role in inducing vaso-occlusive crises (VOCs) in response ... -
My Birthday Wish for Sickle Cell Patients
I turned 31 on Aug. 20, 2020. That’s 31 years as a sickle cell patient, ... -
Grant Expands SCD Patient Registry of COVID-19 Infections to Include Africa
The SECURE-SCD registry, which is collecting de-identified data on individuals with sickle cell disease (SCD) ... -
What Chadwick Boseman Taught Me About Perseverance
Death always is a sobering thing to process. Hearing about someone’s untimely passing — whether ... -
Sickle Cell Awareness Month Events Underway Across US
September is National Sickle Cell Awareness Month, set aside annually to bring attention to the ... -
European Commission Grants Orphan Drug Status to Imara’s IMR-687 for Sickle Cell Disease
The European Commission has granted orphan drug designation to IMR-687, Imara’s potential new therapy for ... -
Breaking the Habit of Assuming the Worst in Others
Looking at my diagnosis in a positive light doesn’t come naturally to me. I usually ... -
EDIT-301, Experimental Cell Therapy for SCD, Earns Rare Pediatric Disease Designation
The U.S. Food and Drug Administration (FDA) has given rare pediatric disease designation to EDIT-301, ... -
Medical Marijuana Reduces Pain and Hospital Visits in SCD Patients
Use of medical marijuana to ease pain is associated with fewer visits to the hospital ... -
Sickle Cell Patients Should Have Medical Exemption in the UK
In the U.K., the National Health Service maintains a medical exemption list with conditions that ... -
First Sickle Cell Patient Dosed in Phase 2b Trial of Imara’s IMR-687
A Phase 2b clinical trial that is investigating the safety and effectiveness of IMR-687 in ... -
EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US
The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to ... -
Rolling With the Punches
Life can change so unexpectedly, especially when dealing with the unpredictability of sickle cell. Sometimes ... -
Sickle Cell Oral Treatment Xromi Approved in Scotland for Ages 2–9
The Scottish Medicines Consortium (SMC) has approved the use of Xromi (hydroxycarbamide), a liquid form ... -
Parallel, Pitt Team Up to Study Medical Marijuana for Treating SCD Symptoms
The cannabis company Parallel, which aims to improve quality of life with its products, has launched ... -
What Not to Say to Someone With Sickle Cell
Have you ever informed somebody of your diagnosis and found their response to be a ... -
Nearly $1M Grant to Support Work Into Reactivating Fetal Hemoglobin
The National Heart, Lung, and Blood Institute (NHLBI) awarded nearly $1 million to Athena Starlard-Davenport, ... -
So, What Is Sickle Cell Disease Again?
In the U.K., approximately 15,000 people have sickle cell disorder and approximately 240,000 have the ... -
‘Be The Match’ Urges Blacks to Join Donor Registry for Sickle Cell Patients
With a stem cell transplant (SCT) being a potentially curative treatment for sickle cell disease (SCD), a ... -
Distress Evident in Parents Caring for Teens with Sickle Cell, Survey Finds
Emotional distress affected nearly half of the parents caring for adolescents with sickle cell disease in ... -
How Lockdown Has Affected My Weight
During this period I have heard a lot of people complain about unwanted weight gain, ... -
CHMP Gives Positive Opinion to Adakveo for Treating Vaso-occlusive Crises in SCD Patients
A European Medicines Agency (EMA) committee has adopted a positive opinion on Novartis‘s Adakveo (crizanlizumab) for ... -
Emergency Care Often Not Ideal for Sickle Cell Patients in US, Survey Finds
Most people with sickle cell disease (SCD) in the U.S. are satisfied with their primary ... -
A Message to Parents Expecting a Child With Sickle Cell
I recently spoke to a mother who gave birth to a child with sickle cell. ... -
Cannabis Seen to Help Ease Pain in Sickle Cell Patients in Small Study
Cannabis could be a safe and potentially effective way of easing chronic pain in people ... -
Staying at Home Has Helped Me Follow a Healthier Routine
Living in lockdown the past few months has been quite an experience. If you had ... -
Umbilical Cord Blood Transplant Treats Children With Genetic Disorders, Trial Finds
An umbilical cord blood transplant, coupled with a reduced intensity conditioning (RIC) regimen, can safely and effectively ... -
Avoid Using the Internet to Self-diagnose
The internet has been a blessing in many of our lives. It is an extremely ... -
Stem Cell Transplant from Unrelated Donors Holds Promise for SCD Patients, Study Suggests
Blood stem cell transplant from unrelated donors is a feasible strategy in people with sickle ... -
Bronchodilators to Treat Asthma May Worsen Youngsters’ SCD, Study Suggests
Bronchodilators used to treat asthma could worsen sickle cell disease (SCD) in children and teens, ... -
Heat Waves Can Exacerbate My Sickle Cell Symptoms
We recently experienced a heat wave here in the U.K., where temperatures climbed as high ... -
Potential Oral DMT Showing Safety and Signs of Efficacy in Phase 1 Trial
FT-4202 — an investigational treatment for sickle cell disease (SCD) — has a favorable safety profile ... -
7th Annual Walk With the Stars & Move-A-Thon Kicks Off July 11 to Fight SCD
While it will be entirely virtual this year, the 7th Annual Walk With the Stars ... -
Finding My ‘New Normal’ as Lockdown Restrictions Ease
It has started … the period I have been uncertain about. Restrictions are starting to ... -
GBT to Request Oxbryta’s Approval in Europe for SCD Patients 12 and Older
Global Blood Therapeutics (GBT) plans to submit a marketing authorization application to the European Medicines ... -
Mitapivat Safely Increases Hemoglobin Levels in SCD Patients, Phase 1 Trial Shows
Mitapivat (AG-348) safely increased the amount of hemoglobin and changed the levels of biomarkers of ... -
On World Blood Donor Day, I Thanked Blood Donors Everywhere
We celebrated World Blood Donor Day on June 14. It reminded me that blood donors ... -
First SCD Patient Treated With CTX001 Remains VOCs-free After 9 Months
The first patient with severe sickle cell disease (SCD) who received a single infusion of ... -
Here Are Ways to Help Friends with Sickle Cell Disease
Whenever I meet new people and tell them I have sickle cell disease (and explain ... -
World Sickle Cell Day Celebrates Patients, Raises Awareness
Supporters are poised to mark World Sickle Cell Day, observed each June 19 to raise ... -
Let’s Celebrate World Sickle Cell Day by Focusing on Our Achievements
June 19 is World Sickle Cell Day, and I have been reflecting a lot on ... -
Bluebird Bio Presents Data from LentiGlobin Gene Therapy Trial
Bluebird Bio’s investigational gene therapy LentiGlobin safely prevents serious vaso-occlusive crises and acute chest syndrome in ... -
Listening to Sickle Cell Patients Is Crucial During a Crisis
I recently was admitted to the hospital during a sickle cell crisis and because of ... -
GBT to Request That Oxbryta Treat Children Starting at Age 4
Global Blood Therapeutics (GBT) will ask the U.S. Food and Drug Administration (FDA) to allow children with sickle ... -
Sickle Cell Takes a Toll on My Veins
Small veins and sickle cell are never a good mix. As luck would have it, ... -
Sickle Cell Society Survey Looks to Better Understand Pandemic’s Affect
The Sickle Cell Society of the U.K. has created a survey to gather feedback from people with sickle ... -
Don’t Take Good Health for Granted
Dealing with sickle cell disease is a constant battle. In every moment, I either am ... -
IMR-687 Safely Increases Fetal Hemoglobin Levels in SCD Patients, Phase 2a Trial Shows
IMR-687 safely increases the levels of fetal hemoglobin and the number of fetal hemoglobin-producing cells ... -
COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say
While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for ... -
Smartphone App May Help Assess Anemia Using Eyelid Pictures
A smartphone app that measures hemoglobin levels using pictures of a person’s inner eyelid may ... -
Vitamin D Supplements May Reduce Pain-related Emergency Room Visits in Children with SCD
Taking vitamin D supplements may lower the number of visits to the emergency department due to vaso-occlusive ... -
What’s Inside My Medicine Cabinet?
Living with sickle cell disease, I rely on several medications and treatments for various situations. ... -
Partnership to Ensure Supply of LentiGlobin, Potential Gene Therapy
Bluebird Bio extended a partnership with two divisions of Hitachi Chemical for the long-term development and manufacturing ... -
How My Parents Set Me Up for Success in Sickle Cell Management
My parents knew they were carriers of the sickle cell trait before I was born, ... -
Sickle Cell Disease Association, Hemanext Join to Support Programs
Hemanext, a medical technology company, is working with the Sickle Cell Disease Association of America (SCDAA) to ... -
Agios to Decide Next Steps for Mitapivat as SCD Treatment by Mid-year
Agios Pharmaceuticals announced that, despite the current COVID-19 outbreak, it remains on track to decide by mid-year next ... -
What Does a Sickle Cell Crisis Feel Like?
One of the most widely discussed symptoms of sickle cell anemia is a sickle cell ... -
Gene-editing Therapy CTX001 Granted RMAT Status by FDA for Severe SCD
The U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to ... -
Sickle Cell Association Issues COVID-19 Guidelines for US Reopening
An advisory committee of the Sickle Cell Disease Association of America is recommending that people with sickle ... -
How I Prepared for the Intense ‘1,000 Reps Challenge’
My activity levels have steeply dipped because I’ve been restricted to my house. I’m unable ... -
Rice University Gets $2.45M NIH Grant to Improve CRISPR/Cas9 Treatment for SCD
The U.S. National Institutes of Health (NIH) has awarded researchers at Rice University a four-year, ... -
APCER Report Confirms Safety of Sickle Cell Therapy Endari
APCER Life Sciences has released a report confirming the safety profile of Endari (L-glutamine), according ... -
‘What’s Wrong with You Now?’
“What’s wrong with you now?” I am all too familiar with that phrase. From a ... -
Fera Requests FDA Orphan Drug Designation for Naproxcinod for SCD
Fera Pharmaceuticals submitted an application to the U.S. Food and Drug Administration (FDA) requesting orphan ... -
Case Researchers Awarded $3.7 Million to Test Emerging SCD Gene Therapies
A team at the Case Western Reserve University has been awarded up to $3.7 million ... -
Keeping Safe During a Lockdown
In the early days of this outbreak, only a couple of media outlets nonchalantly reported ... -
Sickle Cell, Darwinism, and COVID-19: Is My Life Worth Less Than Yours?
“Survival of the fittest” is a phrase I have come to loathe. This manner of ... -
FDA Grants Orphan Drug Designation to FT-4202 for Sickle Cell Treatment
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Forma Therapeutics‘ FT-4202 ... -
HealthWell Foundation Offering Help with Sickle Cell Treatment Costs
The nonprofit HealthWell Foundation launched a new fund to help people with sickle cell disease afford ... -
The Importance of Practicing Gratitude
The intentional practice of gratitude is an essential life skill that’s helped me endure the ... -
Phase 2 Trial of Olinciguat, Oral Sickle Cell Therapy, Completely Enrolled
The Phase 2 STRONG-SCD trial evaluating olinciguat as a potential oral therapy for sickle cell ... -
Sickle Cell Disease Association of America Extends COVID-19 Emergency Fundraiser
The Sickle Cell Disease Association of America (SCDAA) has announced the extension of its ongoing ... -
Self-isolation Prompts a Range of Emotions Requiring Adjustments
I have been self-isolating since March 19, and already I have been through a series ... -
GBT Donating $250,000 to Nonprofits Aiding Sickle Cell Community
Global Blood Therapeutics (GBT) is giving $250,000 to support the U.S. sickle cell disease (SCD) community ... -
During Lockdown, It’s OK to Rest, Too
I was hospitalized on March 3 with a sickle cell crisis and discharged 12 days ... -
Phase 1/2 Trial of Gene-editing Therapy for SCD Cleared by FDA
The U.S. Food and Drug Administration (FDA) has cleared the start of a Phase 1/2 ... -
We Need Credible Information During the Pandemic, Not Dangerous Myths
As a sickle cell advocate, many people have come to me for advice, especially in ... -
The Isolation Diaries
On March 16, 2020, United Kingdom Prime Minister Boris Johnson announced that in response to ... -
Inflammation Markers Identify Sickle Cell Patients at Risk for Worse Outcomes, Study Suggests
High blood levels of two pro-inflammatory molecules, called interleukin-6 (IL-6) and interleukin-8 (IL-8), are linked ... -
Availability of Endari Not Affected by COVID-19 Pandemic, Emmaus Says
The supply of the sickle cell disease (SCD) medication Endari (L-glutamine) will not be affected ... -
Here’s My List of Things to Do While Social Distancing
The spread of the coronavirus means that those of us with sickle cell disease need ... -
GBT Supports Sickle Cell Patients During COVID-19 Pandemic
Global Blood Therapeutics (GBT) is taking proactive measures to support public health efforts and sickle ...