Sickle Cell Disease News

March 4, 2021March 4, 2021

VEST Lowers Duration of Painful SCD Crises

Vascular electrical stimulation therapy (VEST), a form of local electrical stimulation applied to the skin, can lower the duration of painful vaso-occlusive crises (VOCs) in patients with sickle cell disease ... Read more

March 2, 2021March 2, 2021

Stem Cell Transplant, Gene Therapy in SCD Tied to Improved Quality of Life

Sickle Cell Disease News

The quality of life of people with sickle cell disease (SCD) and transfusion-dependent thalassemia (TDT) — two inherited blood disorders — tends to improve if they receive a stem cell ... Read more

February 25, 2021February 25, 2021

Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion

COVID-19 News, Sickle Cell Disease News

Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a young age, he also learned ... Read more

February 24, 2021February 24, 2021

Rare Disease Day at NIH, Set for March 1, Growing Year by Year

COVID-19 News, Sickle Cell Disease News

Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a ... Read more

February 23, 2021February 23, 2021

Gates Foundation, Novartis Working on Affordable Gene Therapy for Sickle Cell

Sickle Cell Disease News

Novartis and the Bill and Melinda Gates Foundation are collaborating to develop a single-shot gene therapy that would cure sickle cell disease (SCD) without the need to engineer cells outside ... Read more

February 19, 2021February 19, 2021

Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients

Sickle Cell Disease News

People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare ... Read more

February 18, 2021February 18, 2021

Bluebird Bio Pauses Trials of Potential Gene Therapy LentiGlobin

Sickle Cell Disease News

Bluebird Bio has paused two clinical trials of LentiGlobin, its gene therapy candidate for sickle cell disease (SCD), after learning that a patient who received treatment more than five years ... Read more

February 16, 2021February 16, 2021

Alternatives to Opioids Seen to Ease Pain, Raise Serotonin Levels in Early Study

COVID-19 News, Sickle Cell Disease News

A good diet and companionship, or treatment with an antidepressant called duloxetine was seen to significantly lessen pain in a mouse model of sickle cell disease (SCD), a study reports. Notably, ... Read more

February 11, 2021February 11, 2021

Oxbryta Can Ease or Fully Heal Leg Ulcers, Trial Analysis Shows

Sickle Cell Disease News

Oxbryta (voxelotor) can help ease, or fully heal, painful leg ulcers in people with sickle cell disease (SCD), according to a post-hoc analysis of a Phase 3 trial. These promising ... Read more

February 10, 2021February 10, 2021

Pandemic Won’t Stop Rare Disease Day on Feb. 28

COVID-19 News, Sickle Cell Disease News

Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness about rare diseases and the ... Read more

February 9, 2021February 9, 2021

NHLBI Challenges Students to Create Innovative Tools to Raise Awareness

Sickle Cell Disease News

A new National Heart, Lung, and Blood Institute (NHLBI) challenge will award a first prize of $25,000 to a team of college or graduate students who develop an innovative tool ... Read more

February 5, 2021February 7, 2021

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns

Sickle Cell Disease News

While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that many concerns — notably out-of-pocket costs ... Read more

February 4, 2021February 4, 2021

Abnormal Flow of Sickled Cells Damages Blood Vessel Walls

Sickle Cell Disease News

Red blood cells in people with sickle cell disease (SCD) flow through blood vessels in a manner that damages the walls, which may explain blood vessel problems and increased inflammation ... Read more

February 2, 2021February 2, 2021

Lonza to Produce Aruvant’s ARU-1801 for Pivotal Trial

Sickle Cell Disease News

A recent manufacturing agreement between Aruvant Sciences and Lonza will advance the development of ARU-1801, an investigational gene therapy for the treatment of sickle cell disease (SCD). Under the agreement, Lonza will ... Read more

January 28, 2021January 28, 2021

EMA Reviews Oxbryta: Therapy Would Be 1st in EU Targeting SCD Root Cause

Sickle Cell Disease News

The European Medicines Agency (EMA) is reviewing an application from Global Blood Therapeutics (GBT) that seeks full marketing approval for the oral therapy Oxbryta (voxelotor) for people with sickle cell ... Read more