GURNEE — Dylan Mudrick looks like other 15-month-old babies. His blue eyes light up when he smiles, and he is at a healthy weight for his age. However, Dylan was diagnosed with a rare disease when he was 4 days old and is now waiting for a liver transplant. When...
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STAMFORD, Conn. — SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, announced today that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for nirogacestat, an oral gamma secretase inhibitor, for the treatment of adults with desmoid tumors. If...
STAMFORD, Conn. — SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, announced today that the U.S. Food and Drug Administration (FDA) has approved OGSIVEO™ (nirogacestat), an oral gamma secretase inhibitor, for the treatment of adult patients with progressing desmoid tumors who require...
Stamford, Conn. — SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, announced that data from the Phase 3 DeFi trial of nirogacestat, an investigational oral gamma secretase inhibitor, in adult patients with progressing desmoid tumors, were...
If approved, mirdametinib is expected to be the first and only therapy in the European Union with marketing authorization for both adults and children with NF1-PN Decision from European Commission expected in the third quarter of 2025 STAMFORD, Conn. — SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused...
SOUTH SAN FRANCISCO, Calif. — Spruce Biosciences, Inc., a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for rare endocrine disorders with significant unmet medical need, today announced completion of enrollment in its CAHmelia-204 clinical trial of tildacerfont for the treatment of adult classic congenital adrenal hyperplasia (CAH)....
SOUTH SAN FRANCISCO, Calif. – Spruce Biosciences, Inc. (Nasdaq: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for neurological disorders with significant unmet medical need, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to tralesinidase alfa enzyme replacement...
LONDON, UK — Spur Therapeutics today announced updated clinical data for avigbagene parvec (FLT201), its Gaucher disease gene therapy candidate, demonstrating clinical benefit and favorable safety maintained for more than two years to date following administration of a single low dose. These data are being presented at the European Society of...
STEVENAGE, UK – FLT201, an experimental gene therapy developed by Spur Therapeutics, has shown sustained clinical benefits for up to 21 months in people with Gaucher disease type 1, according to new data from a clinical trial. The findings, presented at the 28th Annual Meeting of the American Society of...
ALISO VIEJO, Calif. — SpyGlass Pharma™, a privately-held ophthalmic biotechnology company, announced today 1-year follow up data from a first-in-human study of 23 patients with glaucoma or ocular hypertension implanted with SpyGlass’ Intraocular Lens (IOL) based Drug Delivery Platform with bimatoprost at the time of cataract surgery. These data will...