FDA Authorizes Expanded Access Program (EAP) to Help Provide Access to SPG302 for 200 ALS Individuals Ineligible for Clinical Study EAP Supports Real-world Data Collection Concurrent to Ongoing Clinical Studies of SPG302 LOS ANGELES, Calif. — Spinogenix, Inc., a clinical-stage biopharmaceutical company pioneering first-in-class therapeutics that restore synapses to...
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BARCELONA, Spain — SpliceBio, a clinical-stage genetic medicines company pioneering protein splicing to address diseases caused by mutations in large genes, today announced that the first patient has been dosed in the Part B dose-expansion portion of the Phase 1/2 ASTRA clinical trial of SB-007, a dual adeno-associated viral (AAV)...
JERUSALEM, Israel — SpliSense, a clinical-stage biotechnology company developing transformative RNA-based therapies for pulmonary diseases, today announced that the European Medicines Agency (EMA) has granted early Priority Medicines (e-PRIME) designation to SPL84 for the treatment of cystic fibrosis (CF) in patients carrying the 3849+10Kb C>T mutation in the CFTR gene. The e-PRIME designation...
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...
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. (Nasdaq: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for neurological disorders with significant unmet medical need, today announced the successful completion of Type B meetings with the U.S. Food and Drug Administration (FDA or the Agency) regarding its...
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)....