Sickle Cell Disease
A page on conditions in our medical research coverage, part of our health reporting, alongside the 6 reports filed on it so far.
Alnylam defends silencer drugs after
Alnylam defends its RNAi drugs after AstraZeneca's heart disease trial failure, arguing they work even for patients on stabilizer therapies.
T Cell Therapy Treats Atopic Dermatitis
A Lancet comment published August 21, 2026, discusses rezpegaldesleukin, a novel therapy designed to expand regulatory T cells for treating
Revolution Medicines' Rasonque Approved for Pancreatic Cancer
The FDA has approved Rasonque, a new pancreatic cancer drug by Revolution Medicines, marking the first treatment to target a genetic cause of the...
Capricor’s Duchenne Drug Study Highlights the Challenges of Rare Disease Trials
The FDA's recent decision on Capricor’s deramiocel for Duchenne muscular dystrophy underscores the complexities of rare disease clinical trials...
Regeneron's Pasatru Receives FDA Approval for Rare Bone Disease
Regeneron Pharmaceuticals' Pasatru has earned FDA approval for treating fibrodysplasia ossificans progressiva, a rare condition causing abnormal bone...
FDA Clears Bristol Myers Squibb's Oral Multiple Myeloma Therapy, Introducing First-in-Class Agent
The U.S. FDA has approved Bristol Myers Squibb’s oral drug iberdomide, marketed as Zenbexus, for use with daratumumab and dexamethasone in patients...