Tag: syndrome

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…

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…

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Halia Therapeutics Completes Enrollment in Phase 2a Clinical Trial of HT-6184 for Myelodysplastic Syndrome (MDS)

LEHI, Utah, June 4, 2025 /PRNewswire/ -- Halia Therapeutics, a clinical-stage biopharmaceutical…