Acadia Expands DAYBUE STIX Access for Rett Syndrome
San Diego, California, USA, April 7, 2026 Broad U.S. Availability Expands Patient Access In a major advancement for rare...
pediatric neurology
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Praxis Precision Medicines Gains FDA Priority Review Boost
BOSTON, March 30, 2026 Praxis Precision Medicines has achieved a major regulatory milestone as the U.S. Food and Drug...
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...
Merz Submits EMA Application for Pediatric XEOMIN Spasticity Use
FRANKFURT AM MAIN, GERMANY | January 26, 2026 — Merz Therapeutics has completed and submitted a regulatory application to...
FDA Breakthrough Status Advances Rett Syndrome Gene Therapy
DALLAS, Oct. 2, 2025 — The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to TSHA-102,...
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