LentiGlobin, Bluebird Bio’s investigational gene therapy for sickle cell disease (SCD), is unlikely to be the cause of…
Articles by Marisa Wexler, MS
Red blood cells in people with sickle cell disease (SCD) flow through blood vessels in a manner that damages…
The European Medicines Agency (EMA) is reviewing an application from Global Blood Therapeutics (GBT) that seeks full marketing approval…
FT-4202, a potential disease-modifying oral treatment for sickle cell disease (SCD), is well-tolerated in people with SCD and induces…
A team of researchers led by UNC Project-Malawi has been given a one-year, $500,000 grant to build clinical and…
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of…
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like…
FTX-6058, an investigational treatment for sickle cell disease (SCD) and beta-thalassemia being developed by Fulcrum Therapeutics,…
Medications that activate the protein soluble guanylyl cyclase (sGC) may have promise in the treatment of sickle cell…
Using a newly developed assay, researchers identified seven U.S. Food and Drug Administration (FDA)-approved medications that may be effective,…