The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of…
Articles by Marisa Wexler, MS
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,…
The SECURE-SCD registry, which is collecting de-identified data on individuals with sickle cell disease (SCD) who develop…
A European Medicines Agency (EMA) committee has adopted a positive opinion on Novartis‘s Adakveo (crizanlizumab) for the…
Blood stem cell transplant from unrelated donors is a feasible strategy in people with sickle cell disease (SCD), with…
FT-4202 — an investigational treatment for sickle cell disease (SCD) — has a favorable safety profile and the expected…
Global Blood Therapeutics (GBT) plans to submit a marketing authorization application to the European Medicines Agency (EMA)…