News

April 20, 2021April 20, 2021

‘Real Impact’ Grants Help Nonprofits Help People With Blood Disorders

News

Imara has opened applications for its second-annual “Real Impact” grants for community-based nonprofit organizations that support people with rare blood disorders, such as sickle cell disease (SCD) and beta-thalassemia. Through ... Read more

April 16, 2021April 15, 2021

EveryLife Scholarship Fund Open Until May 7 for Adults With Rare Diseases in US

News

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a ... Read more

April 15, 2021April 15, 2021

Partners Seek to Educate About Pediatric Blood Transfusions

News

To inform sickle cell disease (SCD) patients and their caregivers about blood transfusions, which are commonly used to treat the disorder, the Sickle Cell Disease Association of America (SCDAA) is launching ... Read more

April 13, 2021April 13, 2021

Oxbryta Increased Hemoglobin, Reduced Hemolysis in Adults and Adolescents

News

Treatment with oral Oxbryta (voxelotor) leads to rapid and sustained rises in hemoglobin levels, reduces red blood cell destruction (hemolysis), and improves overall health in adolescents and adults with sickle cell disease (SCD), ... Read more

April 8, 2021April 8, 2021

FT-4202 at Higher Dose Safe and Helpful to Patients in Phase 1 Trial

News

A higher 600 mg daily dose of FT-4202, a potential disease-modifying oral treatment for sickle cell disease (SCD), was well-tolerated and safe, and improved several measures of red blood cell ... Read more

April 6, 2021April 6, 2021

GBT Granted Rights to SCD Small Molecule Programs in Deal With Sanofi

News

Global Blood Therapeutics (GBT) has been granted exclusive global rights to two small-molecule research programs for sickle cell disease (SCD) as part of a newly announced agreement between GBT and Sanofi. ... Read more

April 1, 2021April 1, 2021

1st CRISPR Gene Editing Trial Slated to Open in Severe SCD Patients

News

The U.S. Food and Drug Administration approved the start of a first clinical trial of CRISPR_SCD001, the first non-viral and CRISPR/Cas9-based gene editing therapy for sickle cell disease (SCD). Both the ... Read more

April 1, 2021April 1, 2021

Cost of Rare Diseases in US? Nearly $1 Trillion in 2019, EveryLife Foundation Finds

News, syndicated

As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking. But exactly how expensive — in terms of ... Read more

March 30, 2021March 30, 2021

Imara Opens Higher Dose Arms in Phase 2b Trials of IMR-687

News

Imara is opening higher dose arms in two Phase 2b trials assessing the safety and efficacy of IMR-687, its potential therapy for sickle cell disease (SCD) and beta-thalassemia, in patients ... Read more

March 25, 2021March 25, 2021

Graphite Bio Raises $150M to Advance GPH101, Other Gene-editing Therapies

News

Graphite Bio has raised $150 million in Series B funding to expand and advance the clinical development of its pipeline of investigational gene-editing therapies — including GPH101, a potentially curative ... Read more