rare disease treatment – Discover Pharma

Welcome to Discover Pharma! Today is : October 28, 2026

rare disease treatment

Clinical & Regulatory,Pharmaceuticals and therapeutics,Research & Development,Therapeutic Areas
CHMP recommends EU approval of Lynavoy for cholestatic pruritus in PBC
The EMA’s CHMP has recommended EU approval of Lynavoy for [...]

July 24, 2026
Central Nervous System,Clinical & Regulatory,Pharmaceuticals and therapeutics,Research & Development
CHMP recommends approval of NEZGLYAL for rare neurodegenerative disease
The EMA’s CHMP has recommended approval of NEZGLYAL for boys [...]

July 24, 2026
Advanced Therapies,Clinical & Regulatory,Genomics and sequencing,Pharmaceuticals and therapeutics,Research & Development
Atsena receives EMA orphan designation for two inherited retinal disease gene therapies
Atsena Therapeutics has received EMA orphan designation for two gene [...]

July 24, 2026
Clinical & Regulatory,Pharmaceuticals and therapeutics,Therapeutic Areas
FDA accepts Merck NDA for pimicotinib in tenosynovial giant cell tumor
Merck has announced that the US Food and Drug Administration [...]

May 5, 2026
Biotech & Pharma Business,Central Nervous System,Clinical & Regulatory,Therapeutic Areas
Servier acquires Kaerus drug candidate for Fragile X syndrome, the most common genetic cause of autism
Servier has acquired KER-0193 from Kaerus Bioscience, gaining a potential [...]

May 5, 2026
Advanced Therapies,Clinical & Regulatory
UK experts urge Government to appoint Minister for cell and gene therapies
Commission sets out 25 policy recommendations to unlock access and [...]

May 5, 2026

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