#Rare Diseases

Articles (12)

• 🇺🇸 Neurocrine to buy Soleno Therapeutics for $2.9 billion — 06/04/2026 [USA]

  Neurocrine ⁠has ‌offered $53 per Soleno share ⁠held, which represents a premium of about 34% to the stock's last close.

  Related: #Pharmaceutical Acquisition
• 🇺🇸 Neurocrine to acquire Soleno Therapeutics for $2.9 billion — 06/04/2026 [USA] Related: #Pharmaceutical Acquisition
• 🇺🇸 Scientist with rare ALS genetic mutation looks to experimental treatment — 04/04/2026 [USA]

  Every few months for the past three years, Jeff Vierstra has been receiving infusions in his spine that target and disable a mutated gene that made it likely he would develop ALS.

  Related: #Medical Research
• 🇺🇸 Biogen to acquire Apellis Pharmaceuticals for $5.6 billion — 01/04/2026 [USA] Related: #Pharmaceutical Merger
• 🇬🇧 My daughter has childhood dementia and may not live past 16 — 31/03/2026 [United Kingdom]

  Diagnosed just before her fourth birthday, Sophia, now 15, can no longer speak and cannot walk unaided.

  Related: #Family Care, #Medical Challenges, #Child Health
• 🇺🇸 Every Cure’s drug repurposing strategy isn't what you think, and it could change rare disease treatment — 27/03/2026 [USA]

  When David Fajgenbaum and his colleagues launched the drug repurposing non-profit Every Cure, they chose a path that challenged the traditional research model.

  Related: #Drug Repurposing
• 🇺🇸 Mind the Rarities: Can Rare Skin Diseases Be Reliably Diagnosed via Diagnostic Reasoning? — 20/03/2026 [USA]

  arXiv:2603.18418v1 Announce Type: cross Abstract: Large vision-language models (LVLMs) demonstrate strong performance in dermatology; however, evaluating diagnostic reasoning for rare conditions rema...

  Related: #Medical Diagnosis
• 🇺🇸 Knowledge Graph Extraction from Biomedical Literature for Alkaptonuria Rare Disease — 18/03/2026 [USA]

  arXiv:2603.15711v1 Announce Type: new Abstract: Alkaptonuria (AKU) is an ultra-rare autosomal recessive metabolic disorder caused by mutations in the HGD (Homogentisate 1,2-Dioxygenase) gene, leading...

  Related: #Biomedical Research, #Data Extraction
• 🇺🇸 Savara receives FDA priority review for rare lung disease drug — 13/03/2026 [USA] Related: #FDA Approval
• 🇺🇸 Ultragenyx reports positive Phase 3 data for OTC gene therapy — 12/03/2026 [USA] Related: #Gene Therapy, #Clinical Trials
• 🇺🇸 PTC Therapeutics at Leerink Conference: Strategic Growth in PKU Market — 11/03/2026 [USA] Related: #Biopharmaceuticals
• 🇺🇸 Ultragenyx at Barclays Conference: Strategic Insights on Rare Diseases — 11/03/2026 [USA] Related: #Biotech