News

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 Patient and Family Forum. The interactive, patient-focused forum will be held online June 26-27. The deadline to apply for…

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 indicate. The results observed in this initial group of patients support the efficacy of Forma Therapeutics‘ investigational medication, as…

Sickle Cell Disease News brought you daily coverage of key findings, treatment developments, clinical trials, and other important events related to sickle cell disease (SCD) throughout 2020, a year marked by the COVID-19 pandemic. As a…

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 began last month and is aimed at giving the Army a snapshot of how SCT has affected its ranks, and…

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), according to updated data from the CLIMB-SCD-121 Phase 1/2 trial. CTX001 is being jointly developed by CRISPR…

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 of charge. Patients who currently lack insurance coverage or financial means to afford treatment may reach out to Endari’s…

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 case report suggests. These findings are particularly important during the ongoing pandemic in the U.S., in which rising blood…

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 (SCD), new analyses of the RESET clinical trial show. The data further support “the potential benefits of…

Global Blood Therapeutics (GBT) has launched an early access program for voxelotor in Europe and other regions outside the U.S. to allow people with hemolytic anemia associated with sickle cell disease (SCD) to have access to treatment. The early access program will be open to those…

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) has launched an initiative across the U.S. Its goal is to establish a Rare Disease Advisory Council (RDAC)…