syndrome

Nacuity Pharmaceuticals Announces Positive Data from Clinical Trial Evaluating NPI-001 to Treat Retinitis Pigmentosa Associated with Usher Syndrome

September 11, 2025 09:00 ET  | Source: Nacuity Pharmaceuticals, Inc. NPI-001 shows more than 50% reduction in photoreceptor loss caused…

2 weeks ago

Servier acquires potential treatment for Fragile X syndrome, the most common genetic cause of autism spectrum disorder

Servier acquires KER-0193, a potential treatment for Fragile X syndrome, developed by Kaerus Bioscience, a Medicxi companyKER-0193, a novel, orally bioavailable…

2 weeks ago

UCB presents latest research and clinical advancement across leading epilepsy portfolio at International Epilepsy Congress

26 scientific abstracts, including two oral presentations, demonstrate UCB's ongoing commitment to advancing research for people living with epilepsiesData include an…

4 weeks ago

Galapagos NV Announces U.S. FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to GLPG5101 for the Treatment of Relapsed/Refractory Mantle Cell Lymphoma

Mechelen, Belgium; August 6, 2025, 7:30 CET; regulated information – inside information – Galapagos NV (Euronext & NASDAQ: GLPG) today…

2 months ago

Ultragenyx Receives Complete Response Letter from FDA for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)

July 11, 2025 16:30 ET  | Source: Ultragenyx Pharmaceutical Inc. Complete Response Letter (CRL) cited specific chemistry, manufacturing and controls…

2 months ago