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Virginia Tech Student Awarded NIH Grant for Research Into Rett Protein
A first-generation university student has been awarded a six-year, $466,669 grant from the National Institutes ...
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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 ... -
Shape Therapeutics Raises $112M to Develop RNA-based Gene Therapies
Shape Therapeutics has raised $112 million in financing to support work into developing its portfolio ... -
Nominations Open for 2022 Eurordis Black Pearl Awards
Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. ... -
Rett Researcher Discovers Molecular Mechanism of MECP2 Binding
The molecular mechanism by which the protein MeCP2 recognizes and binds to CA repeats — ... -
Mouse Study Suggests Need to Test Therapeutic Approaches Across Rett Models
Treatment with a protein called CNF1 produced different results in two different mouse models of ... -
Virtual Avatar Improved Attention and Memory
Using a virtual avatar to guide users during attention and memory tasks significantly improves the ... -
MeCP2 Protein’s Lack May Be Driver of Neuroinflammation in Patients
The lack of a working MeCP2 protein — the most common cause of Rett syndrome ... -
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 ... -
MicroRNAs Improve Behavior, Motor Function in Rett Mouse Model
The use of microRNA molecules that targeted a protein receptor important for nerve cell growth ... -
EveryLife Introduces First of Kind ‘Roadmap’ to ICD Codes
To help patient advocacy leaders and their partners better understand how global health statistics codes ... -
Latest Trial Results of Anavex 2-73 ‘Very Promising’
Clinical improvements with Anavex 2-73 (blarcamesine) correlated with activation of the SIGMAR1 gene, which provides instructions ... -
Caregivers Asked to Help in Adapting Tool ‘Critical’ to Therapy Trials
The nonprofit Rett Syndrome Research Trust (RSRT) and Duke University’s Center for Health Measurement are asking Rett ... -
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 ... -
Alcyone Therapeutics Opens, Focus on Gene Therapy for Rett Syndrome
Alcyone Therapeutics, a new company focused on developing gene therapies for disorders that affect the ... -
Researchers Win $1.1M to Switch On Backup Copy of MECP2
The Rett Syndrome Research Trust (RSRT) has given two researchers $1.1 million to collaborate on ... -
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 ... -
Nocturnal Teeth Grinding Linked to Frequent Snoring
Bruxism, also known as teeth grinding and jaw clenching, during the night is associated significantly ... -
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 ... -
Potential New Therapeutic Targets Identified in Preclinical Study
Targeting a biological pathway regulated by MECP2 — the gene mutated in most cases of Rett ... -
MeCP2-boosting Approaches Led to Toxic Events in Mice
Providing a working version of MECP2 — the gene mutated in most cases of Rett syndrome — to ... -
Changes in Gut Microbiota Associated With Disease Severity
The richness and diversity of the gut’s microbial community, or microbiota, are reduced significantly after ... -
Mouse Study Alerts Potential Gene Therapy Issues in Female Patients
A gene therapy-like approach that provided a full-length MECP2 — the gene mutated in most ... -
Disease Severity Tied to Two Neuroprostanes
Elevated levels of two neuroprostanes — markers of oxidative stress, a result of insufficient antioxidant ... -
Parents of Rett Patients Report Reduced Quality of Life, Family Functioning
According to their parents, children with Rett syndrome have diminished quality of life (QoL) across ... -
Potential Gene Therapy TSHA-102 Does Well in Animal Study
TSHA-102, an experimental gene therapy for Rett syndrome, safely and effectively normalized levels of MeCP2 — ... -
15 Clinics in 14 U.S. States Part of Center of Excellence Network for Rett
The International Rett Syndrome Foundation (IRSF) has designated 15 clinics across the U.S. as members ... -
Wearable Devices Capture Changes in Emotions, Behavior in Patients
Skin conductance — the electrical activity on the surface of the skin in response to ... -
Low Dietary Protein Linked to Growth Deficits in Taiwan
A study suggests lower intake of dietary protein is associated with more severe growth deficits ... -
Daytime Teeth Grinding Means More Dental Work
People with Rett syndrome who grind their teeth during the day had a higher incidence ... -
Quick Approval for COVID Vaccines a Concern, Rare Disease Patients Say
More than half of the people with rare diseases and their caregivers, asked in a ... -
Early Treatment Reverses Cell Defects, Improves Survival in Mice
Early administration of a compound intended to enhance signals between brain cells lessened disease severity, ... -
Social Family Activities Engage Rett Patients More Than Daily Routines
Girls and young women with Rett syndrome have a high level of both participation and ... -
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 ... -
Board Recommends All Anavex 2-73 Trials Proceed
An independent monitoring board has recommended that all three ongoing clinical trials of Anavex 2-73 (blarcamesine), ... -
MeCP Protein’s Ability to Interact With DNA May Be Crucial for Brain Health
MeCP2, the protein missing or defective in most people with Rett syndrome, contains a specific ... -
LAVENDER Phase 3 Trial of Trofinetide Enrolling at New Sites
Acadia Pharmaceuticals is enrolling participants at four new clinical sites in its Phase 3 LAVENDER trial ... -
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 ... -
COVID-19 Vaccine Compatible with Trial Testing of Rett Gene Therapy
People with Rett syndrome can receive the COVID-19 vaccine from Johnson & Johnson and still ... -
Skills Training Given Before Symptoms Evident May Help Rett Children
Early, intensive training in motor and memory skills before symptoms develop may delay disease progression ... -
Grant Will Fund Study of Breathing Difficulties
A $2.1 million grant will fund a study investigating if — and how — cells ... -
Potential Gene Therapy for Rett Syndrome in the Works
A gene therapy aimed at slowing disease progression and improving movement, coordination and communication in ... -
Balance of Cohesin Proteins Appear Central to X Chromosome Activity
A fine balance of proteins called cohesins is important for the normal inactivation of the ... -
COVID-19 Economic Stimulus Legislation Welcomed by The Arc
The Arc and its advocates are welcoming the U.S. Congress’ decision to pass COVID-19-related economic stimulus ... -
Taysha, Yale to Advance Gene Therapy Program
Taysha Gene Therapies has established a new research partnership with Yale University to advance its ... -
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 ... -
The Arc Awarded Comcast NBCUniversal Grant for Special Education Initiative
A $200,000 Comcast NBCUniversal grant has been awarded to The Arc to increase access to ... -
Study Identifies Potential Blood-based Biomarkers, Paths for Treatment
Researchers have identified a set of RNA molecules that are dysregulated in Rett syndrome and ... -
Teeth Grinding Is Most Common Dental Problem
Bruxism, also known as teeth grinding and jaw clenching, is the most common oral manifestation ... -
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 ... -
Cerebellum Dysfunction May Contribute to Rett Motor Problems
Impairments in the cerebellum — a brain region responsible for controlling balance and movement coordination ... -
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 ... -
DiamiR Receives Patent Allowance for Rett MicroRNA Biomarker
The United States Patent and Trademark Office (USPTO) has issued an allowance for a patent ... -
Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients
People with rare disorders have a worse healthcare experience than those affected by chronic diseases, ... -
Novartis to Seek Approval by Year’s End to Test Gene Therapy Candidate
Novartis Gene Therapies is planning to submit an investigational new drug application (IND) for OAV201, its ... -
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 ... -
Electrical Responses in Brain to Stimuli May Be Marker of Rett Severity
People with Rett syndrome have significantly weaker electrical responses in the brain to visual and ... -
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 ... -
Iron Deposits in Brain May Contribute to Dystonia in Rett Patients
Dystonia, or excessive involuntary muscle contractions, are linked in Rett syndrome with abnormal iron accumulation in ... -
Gilenya Fails to Ease Rett Symptoms in Phase 1/2 Trial
Despite being safe, one year of treatment with Gilenya (fingolimod) in young girls with Rett syndrome ... -
IQSEC2‐related Intellectual Disability Should Be Part of Rett Spectrum
Patients with milder forms of IQSEC2‐related intellectual disability experience symptoms and have an overall clinical ... -
Small Molecule Aids Motor Skills, Breathing in Rett Mouse Model
Use of PTX-BD4-3, a small molecule that activates a protein receptor involved in a pathway ... -
Girls With Rett Have Higher Rate of Menstrual Seizures, Study Says
Girls and women with Rett syndrome may have increased rate of menstrual seizures, also known ... -
Distinct Immune Protein Pattern Found in Rett Patients in Small Study
In people with Rett syndrome, cells known as fibroblasts have a distinct protein production profile ... -
Anavex 2-73 Seen to Safely Ease Rett Severity in Women in Phase 2 Trial
Anavex 2-73 (blarcamesine), an investigational treatment for Rett syndrome, lessened the disease’s characteristic behavioral features, ... -
Early Work Using Lab-grown ‘Mini-brains’ Identifies 2 Potential Rett Therapies
Scientists have identified two compounds that were able to rescue nerve cell defects caused by ... -
Anavex 2-73 Treatment for Rett to Receive New US Patent Broadening Coverage
Anavex Life Sciences has received a Notice of Allowance from the U.S. Patent and Trademark ... -
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 ... -
Reverse Rett Hosts Dec. 5 Virtual Big Night In Fundraiser, Benefitting Research
Viewers from around the world are invited to join in the virtual Reverse Rett Big ... -
GW Stops ARCH Phase 3 Trial Testing Epidiolex in Children, Adolescents, Due to COVID-19
Due to the unprecedented challenges caused by the ongoing COVID-19 pandemic, GW Pharmaceutics has stopped the ... -
Adults With Rett Require Tailored Care Program to Manage Symptoms, Study Says
The transition into adulthood of women with Rett syndrome warrants a tailored program and a ... -
Rett Animal Model Seen to Share Patients’ Complex Learning Difficulties
Problems with skilled learning tasks that involve motor function and making sense of sounds — ... -
Taysha Gene Therapies, Catalent Partner to Advance Gene Therapies for Rett, Other Disorders
Taysha Gene Therapies and Catalent have established a partnership to advance Taysha’s gene therapies for neurological disorders, ... -
‘Band of Mothers’ Connect to Raise Awareness About Boys With Rett
Of the people who know about Rett syndrome, many think it affects only females. But families ... -
Rett Syndrome Research Trust Seeks Families for Global Registry
As the Rett Syndrome Research Trust (RSRT) prepares to launch a global database of caregiver-provided ... -
Antidepressant Mirtazapine Helps Preserve Motor Function in Rett Patients and Mice, Study Finds
Treatment with the antidepressant mirtazapine preserves motor and respiratory function, as well as social behavior, ... -
FDA Acts to Support Development of Potential Gene Therapy, TSHA-102
The U.S. Food and Drug Administration (FDA) has granted orphan drug and rare pediatric disease designations to ... -
New KIF1A Gene Mutation Linked to Rett, Ends One Girl’s 15-year Diagnostic Journey
Researchers have identified a new mutation in the KIF1A gene in a girl in Australia that ... -
Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos
Same But Different, a nonprofit U.K. group that uses art for social change, is inviting ... -
Nearly $500K Awarded to Support Search for Rett Syndrome Biomarkers
The National Institute for Neurological Disorders and Stroke, a division of the National Institutes of Health ... -
Events, Activities to Take Place Throughout October, Rett Syndrome Awareness Month
A number of events are in the works to mark Rett Syndrome Awareness Month, observed ... -
Overground or Treadmill Walking Suitable to Assess Gait in Rett Patients, Study Finds
Walking overground or on a treadmill is suitable as a tool to observe and detect ... -
Brain Adenosine System May Be Therapeutic Target in Rett, Mouse Study Reports
Addressing changes in the adenosine system, essential for nerve cell function in the brain, may ... -
Study Reveals Similar Spinal Surgery Outcomes in Rett Syndrome and Cerebral Palsy
Patients with Rett syndrome have similar spinal surgery outcomes to those of people with cerebral ... -
Medical Advisory Board to Set Best-in-Class Care, Rettsyndrome.org Says
Rettsyndrome.org has launched the International Rett Syndrome Foundation Medical Advisory Board, aiming to enhance care ... -
Rett Patients With Milder Mutation Have Poorest Quality of Life, Study Finds
Girls and women with Rett syndrome caused by a mutation usually associated with milder disease ... -
First US-based Anavex 2-73 Trial Completed; Top-line Results Due by Year’s End
A Phase 2 clinical trial evaluating the safety and efficacy of Anavex Life Sciences‘ investigational ... -
Vagus Nerve Stimulation Plus Sound May Improve Auditory Processing in Rett, Study Suggests
Stimulation of the vagus nerve, when paired with sound, could help people with Rett syndrome ... -
Study Links Word Acquisition With Walking Ability in Rett Syndrome
Children with Rett syndrome who can verbalize meaningful words are more likely to be able ... -
AVXS-201, Gene Therapy for Rett, Again Moving Toward Clinical Trial, AveXis Says
Most animal studies needed to support plans to move AVXS-201 — AveXis’ investigational gene therapy for ... -
Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input
Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed ... -
Parents of Rett Children in Spain Struggle With Healthcare Access, Time Management, Study Reports
Parents of children with Rett syndrome face difficulties with healthcare access, time management, bureaucracy, and ... -
Molecular Switch Inactivates X Chromosome, May Provide Insight Into Rett
To better understand X-linked disorders such as Rett syndrome, researchers discovered an enzyme called DCP1A ... -
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 ... -
New Streaming Channel Showcases Rare Disease Films
When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated ... -
DNA Methylation Patterns Before Birth and After May Help in Understanding Rett
Researchers at the Salk Institute for Biological Studies have mapped how DNA methylation — a ... -
Taysha Raises $95M to Help Speed Clinical Development of Gene Therapies
Taysha Gene Therapies received $95 million in funding that will support the clinical development of ... -
Novel Function of MeCP2 Protein Could Be Route to Rett Therapy
Mutations in the MECP2 gene, which lead to most cases of Rett syndrome, prevent the ... -
High Stress Levels Found in 40% of Parents Caring for Rett Child in Italy
About 40% of parents whose daughters have Rett syndrome show clinically high levels of stress, ... -
RNA Editing Repairs Rett Syndrome Mutation in Mouse Model
For the first time, scientists have repaired a mutation in mice that underlies Rett syndrome, ... -
BRD4 Protein May be Therapeutic Target for Rett Syndrome, Study Suggests
By targeting a protein called BRD4, an experimental cancer treatment known as JQ1 reversed cellular abnormalities ... -
National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US
The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease ... -
First Patient Dosed in Anavex-2-73 Trial for Children, Adolescents With Rett
A Phase 2/3 trial evaluating the treatment candidate Anavex-2-73 (blarcamesine) in children and adolescents with Rett ... -
New Discovery on X Chromosome Inactivation May Open Treatment Avenues for Rett Syndrome
Researchers have discovered that early inactivation of the X chromosome is a two-step process regulated ... -
Diabetes Therapy Metformin Restores Brain Energy in Rett Mouse Model
Treatment with the diabetes therapy metformin is able to restore energy production in the brain ... -
US Phase 2 Trial of Anavex 2-73 Surpasses Initial Enrollment Goal
U.S. enrollment for a Phase 2 clinical trial of investigational therapy Anavex 2-73 (blarcamesine) for Rett ... -
Phase 3 Trial of Trofinetide in Girls, Young Women Again Enrolling in US
Acadia Pharmaceuticals is again enrolling in its Phase 3 LAVENDER trial testing trofinetide as a treatment ... -
Language, Attention Gains Seen in Brain Stimulation Plus Cognitive Therapy
Combining conventional cognitive training with transcranial direct current stimulation (tDCS) — a non-invasive, painless brain ... -
Coalition Will Address Racial Disparities in Rare Disease Communities
The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on ... -
Genetic Screening in Mice Points to Potential Therapeutic Targets for Rett
Several genes involved in lipid (fat) metabolism, nerve cell communication, and DNA damage response may ... -
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 ... -
Blocking P2X7 Receptors May Be Therapeutic Target for Rett
Blocking the activity of pro-inflammatory P2X7 receptors — a class of receptors found in immune ... -
Scientists Who Discovered Gene Link with Rett Syndrome Awarded 2020 Brain Prize
The scientists who discovered the MECP2 gene and its role in Rett syndrome have been ... -
Taysha Raises $30M, Partners with UT Southwestern to Develop Gene Therapies for Rett, Other Disorders
Taysha Gene Therapies has raised $30 million and established a partnership with UT Southwestern Medical ... -
Newron Terminates Sarizotan Development Due to STARS Trial Failure
Treatment with sarizotan failed to reduce apnea episodes (breathing stops) while awake compared to a ... -
Therapy Using ‘Unstable’ Gene Seen to Help Mice in Rett Model
A form of gene therapy based on providing an unstable version of the MECP2 gene ... -
Altered Nerve Cell Communication Tied to Motor Difficulties of Rett
Problems with nerve cell communication and adaptation in the brain’s motor cortex is associated with ... -
Differences in Metabolism May Aid Rett Syndrome Characterization and Treatment
A comprehensive metabolic analysis uncovered significantly different levels of 66 molecules in people with Rett ... -
Rett Syndrome Trial of Oral Anavex 2-73 to Continue with at-Home Visits
Anavex Life Sciences announced plans to continue to screen and enroll patients into its active ... -
Researchers Shed Light on Link Between IGF-1 and Potential Benefits in Rett Syndrome
Treatment with insulin-like growth factor 1 (IGF-1) eases symptoms of Rett syndrome by suppressing the ... -
Amicus Opens Gene Therapy Center Focused on Rett Syndrome, Other Rare Diseases
Amicus Therapeutics has opened its Global Research and Gene Therapy Center of Excellence that will ... -
Newron Pharma Receives FDA Communication on STARS Trial; Results Expected Soon
Newron Pharmaceuticals has received the final minutes from a meeting with the U.S. Food and ... -
Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds
The number of treatments for children with rare diseases has grown over the past decade, according ... -
MECP2 Mutation Type Predicts Rett Syndrome Bone Disease Severity, Study Finds
The type of mutation in the MECP2 gene in people with Rett syndrome is a ... -
FDA Grants Rare Pediatric Disease Designation to Trofinetide for Rett Syndrome
The U.S. Food and Drug Administration (FDA) granted rare pediatric disease designation to trofinetide, a potential ... -
Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare
In recognition of Rare Disease Day Feb. 29, BioNews Services launched a social media campaign ... -
Restoring Wnt6 Protein Eases Behavioral, Motor Impairment, Mouse Study Suggests
Restoring the levels of Wnt6, a signaling molecule involved in brain function, eased motor and behavioral ...