News

January 14, 2021January 14, 2021

BCH-BB694 Gene Therapy Safely Treating Severe SCD Patients in Pilot Trial

News

BCH-BB694 — an experimental gene therapy targeting the BCL11A gene — safely increased the levels of fetal hemoglobin and prevented disease-associated complications in six people with severe sickle cell ... Read more

January 12, 2021January 12, 2021

Oral IMR-687 Seen to Lessen VOCs, Pain Crises in Patients in Phase 2a Trial

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Six months of treatment with oral IMR-687 safely and effectively reduced the number of vaso-occlusive crises (VOCs) and other disease-associated pain crises in adults with sickle cell disease (SCD), updated ... Read more

January 11, 2021January 11, 2021

NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum

News, syndicated

The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD ... Read more

January 7, 2021January 7, 2021

Oral FT-4202 Safe, Found to Improve Red Blood Cell Function in SCD

News

FT-4202, a potential disease-modifying oral treatment for sickle cell disease (SCD), is well-tolerated in people with SCD and induces improvements in several red blood cell parameters, early clinical trial data ... Read more

January 5, 2021January 5, 2021

US Army Begins Testing Recruits for Sickle Cell Trait

News

To identify at-risk service members, the U.S. Army has begun testing recruits for the sickle cell trait (SCT), and expects all to be screened within a year. The testing ... Read more

December 17, 2020December 18, 2020

CTX001 Continues to Prevent VOCs in People With Severe Disease, Trial Finding

News

CTX001, an experimental gene-editing cell therapy, safely and effectively increased the levels of fetal hemoglobin and prevented vaso-occlusive crises (VOCs) in three people with severe sickle cell disease (SCD), ... Read more

December 15, 2020December 15, 2020

Emmaus Support Program Gives US Patients Low-cost Access to Endari

News

Emmaus Life Sciences has launched a program that will allow eligible sickle cell disease patients in the U.S. to access its Endari (L-glutamine) medication at low cost or free ... Read more

December 10, 2020December 10, 2020

Oxbryta May Be SCD Blood Transfusions Option in Pandemic, Case Report Finds

News

Treatment with Oxbryta (voxelotor) may be a feasible alternative to blood transfusions for sickle cell disease (SCD) patients who are experiencing respiratory distress and pneumonia related to COVID-19, a ... Read more

December 8, 2020December 8, 2020

SCD Patients Benefit from Early Rivipansel Treatment for VOCs, New Analyses Show

News

Starting treatment with rivipansel (GMI-1070) shortly after vaso-occlusive crisis (VOC) onset significantly shortened hospital stays and the time to opioid discontinuation in children and adults with sickle cell disease ... Read more