Praxis Precision Medicines Reports Strong Elsunersen Trial
Boston, Massachusetts, USA, April 6, 2026 Breakthrough Results in Rare Pediatric Epilepsy In a major advancement for precision neuroscience...
rare disease
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Amgen TEPEZZA Subcutaneous Trial Shows Strong Phase 3 Results
Thousand Oaks, California, USA, April 6, 2026 Phase 3 Success Expands TEPEZZA Treatment Options In a major advancement for...
Affinia Therapeutics Wins Canada Nod for AFTX-201 Trial
Waltham, Massachusetts, USA, April 6, 2026 Health Canada Approval Marks Key Clinical Milestone In a significant development for cardiovascular...
Tenvie Therapeutics Doses First Patient in TNV262 Trial
Brisbane, California, USA, April 6, 2026 First-in-Human Study Targets Inflammation Pathways In a significant milestone for clinical-stage biotech innovation,...
Neurocrine Acquires Soleno to Boost Rare Disease Portfolio
San Diego & Redwood City, California, USA, April 6, 2026 Strategic Acquisition Strengthens Rare Disease Leadership In a major...
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Ultragenyx Advances UX016 Into Clinical Trials for GNEM
NOVATO, Calif., March 30, 2026 Ultragenyx Pharmaceutical Inc. has announced that the U.S. Food and Drug Administration (FDA) has...
Glycomine Advances GLM101 Into Phase 2b Rare Disease Trial
SAN CARLOS, Calif., April 2, 2026 Glycomine, Inc. has announced the successful completion of patient enrollment in its global...
Ultragenyx Advances UX111 Gene Therapy Toward Approval
NOVATO, Calif., April 2, 2026 Ultragenyx Pharmaceutical Inc. has reached a significant regulatory milestone as the U.S. Food and...
Ocugen Completes Dosing in Phase 2/3 Stargardt Trial
MALVERN, Pa., USA — April 1, 2026 Ocugen, Inc. has announced the early completion of dosing in its Phase...
Pasithea Wins FDA Fast Track for PAS-004 Cancer Drug
MIAMI, USA — April 1, 2026 Pasithea Therapeutics Corp. has announced that the U.S. Food and Drug Administration (FDA)...
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