A mother whose daughter was born with a rare chromosome disorder meaning she is non-verbal and has scoliosis along with a ...
Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into ...
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 ...
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 ...
Alexander Bryant, 39, was diagnosed with stage four tonsil cancer last December and was recently told it is non-curable, and ...
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 ...
Following a single subcutaneous 240 mg dose, WVE-007 (INHBE GalNAc-siRNA) improved body composition at three months compared ...
Wave Life Sciences Ltd. ( WVE) Discusses Positive Interim Data from INLIGHT Trial of WVE-007 for Obesity December 8, 2025 8:30 AM EST ...
SAB Biotherapeutics, Inc. (Nasdaq: SABS), a clinical-stage biopharmaceutical company developing human anti-thymocyte immunoglobulin (hATG) for type 1 diabetes (T1D) and other autoimmune diseases, ...
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 ...
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