Every face is unique. Genetics helps to determine our features, but sometimes genes have errors which, in early fetal ...

Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into ...

Capricor Therapeutics said on Wednesday its cell therapy for a rare muscular disorder met the main goal of a late-stage study, reviving hopes for U.S. approval after a setback and sending its shares ...

So, Regeneron's revenue rose 1.9% from $3.68 billion in Q2 2025 to $3.75 billion, beating consensus estimate by $160 million ...

On a video call in early September, Sarah Tabrizi first saw the data that she and other researchers studying Huntington’s ...

According to DataM Intelligence, the Orphan Drugs Market Size was USD 223.76 billion in 2023 and is forecasted to soar to USD 486.51 billion by 2032, expanding at a strong CAGR of 9.1% between 2024 ...

Alexander Bryant, 39, was diagnosed with stage four tonsil cancer last December and was recently told it is non-curable, and ...

Mycosis fungoides is the most common subtype, making up the vast majority of CTCL cases. While early-stage disease has an estimated five-year survival rate of 88%, it remains a difficult-to-manage ...

The EMBOLD topline results will be presented at the American Epilepsy Society Annual Meeting at the Georgia World Congress Center, Poster Hall B2, 1.38 from 12:00-2:00 p.m. on Saturday, December 6, ...

Clear dose-response observed, with a robust and clinically meaningful fetal hemoglobin (HbF) induction at the Week 6 timepoint (n=12): mean ...