Fortress Biotech Reports Strong Q3 Growth and Pipeline Progress
MIAMI, FL — November 14, 2025 — Fortress Biotech announced its third-quarter 2025 financial results, highlighting sustained revenue growth,...
rare disease
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Rhythm Pharmaceuticals Presents Four New Datasets at ObesityWeek® 2025
Boston, November 10, 2025 — Rhythm Pharmaceuticals, Inc. has announced the presentation of four pivotal datasets at ObesityWeek® 2025,...
Mirum Pharmaceuticals Presents New Data from LIVMARLI and Volixibat Programs at AASLD 2025
FOSTER CITY, Calif., November 7, 2025 — Mirum Pharmaceuticals, Inc., a biopharmaceutical company dedicated to transforming the lives of...
MavriX Bio Doses First Patient in Landmark ASCEND-AS Gene Therapy Trial
Middleton, Massachusetts – November 6, 2025 – MavriX Bio, a clinical-stage biotechnology company focused on transformative genetic medicines, announced...
Pasithea Activates UAB Clinical Site for PAS-004 NF1 Trial
MIAMI, November 4, 2025: Pasithea Therapeutics Corp. has announced the activation of a new clinical trial site at the...
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FDA Accepts BioMarin’s PALYNZIQ sBLA for Priority Review in Adolescents with PKU
San Rafael, California — October 30, 2025 — BioMarin Pharmaceutical Inc. announced that the U.S. Food and Drug Administration...
Halia Therapeutics’ Ofirnoflast Gains FDA Orphan Drug Status for MDS
LEHI, Utah, Oct. 23, 2025 — Halia Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on therapies targeting the root...
Stealth BioTherapeutics Expands Access with Mito Assist™ and AnovoRx Partnership
NEEDHAM, Mass., October 9, 2025 — Stealth BioTherapeutics Inc. has announced the launch of Mito Assist™, a comprehensive patient...
FDA Breakthrough Status Advances Rett Syndrome Gene Therapy
DALLAS, Oct. 2, 2025 — The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to TSHA-102,...
Sobi Taps Kinaxis Platform to Speed Rare Disease Therapy Supply
OTTAWA, Oct. 2, 2025 — Kinaxis announced that Sobi, a specialist in rare‐disease biopharma, will adopt the Kinaxis Maestro®...
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