Ifetroban Gains FDA Fast Track for DMD Heart Disease
NASHVILLE, Tenn., Feb. 4, 2026 —Cumberland Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration has granted Fast...
rare disease
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Ultragenyx Gene Therapy Shows Durable Gains in MPS IIIA
NOVATO, Calif., February 3, 2026 — Ultragenyx Pharmaceutical Inc. has reported positive long-term clinical data for UX111, an investigational...
Idorsia Presents Long-Term Lucerastat Data in Fabry Disease
Allschwil, Switzerland – January 30, 2026 — Idorsia Ltd. announced it will present long-term lucerastat clinical data and kidney...
Ipsen Halts Phase II FALKON Trial in Ultra-Rare FOP
Paris, France, December 19, 2025 — Ipsen, a global biopharmaceutical company, announced an update on its pivotal Phase II...
Praxis Data Shows Breakthrough Seizure Control Across Epilepsies
BOSTON, MA — December 8, 2025 — Praxis Precision Medicines presented compelling new clinical results at the American Epilepsy...
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Tiakis Advances Tiprelestat into Phase II for Pulmonary Hypertension
Kiel, Germany, December 2, 2025 — Tiakis Biotech has announced that it has successfully completed the required preclinical data...
Tinlarebant Phase 3 Trial Marks Major Breakthrough for STGD1
San Diego, December 1, 2025 — Belite Bio has announced positive topline results from the pivotal global Phase 3...
Regeneron and Tessera Unite to Develop TSRA-196 Therapy
Tarrytown, New York & Somerville, Massachusetts, December 1, 2025 — Regeneron and Tessera Therapeutics have announced a global collaboration...
Koselugo Secures US Approval, Advancing Targeted NF1 Care
Wilmington, Delaware, November 2025 AstraZeneca has announced that Koselugo (selumetinib) has received expanded FDA approval in the United States...
EMA Positive Opinion Grants Orphan Drug Hope for CTCL Treatment
Dateline – November 17 2025 | Lund, Sweden BioInvent International AB (“BioInvent”), a clinical-stage biotech company focused on novel...
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