John B. Sanfilippo & Son

Childhood dementia should be more widely recognised, mother says

An affected mother says she cannot think of a worse diagnosis for a child than childhood dementia.

RARE Stock Up 13% on FDA Approval of Sanfilippo Syndrome Gene Therapy

Ultragenyx jumps 13% after FDA approval of Fayuvi, the first approved treatment for Sanfilippo syndrome Type A, backed by durable clinical data.

Ultragenyx sets $3.95 million price for rare disease gene therapy

Ultragenyx ​on Thursday set a U.S. list price of $3.95 million for its newly approved gene therapy Fayuvi, launching one of the world's most expensive drugs.

Ultragenyx Wins First Sanfilippo Approval As Analyst Keeps Ratings Modest

Abeona Therapeutics® Congratulates Ultragenyx on U.S. FDA Approval of FAYUVI™ Gene Therapy for Sanfilippo Syndrome Type A (MPS IIIA)

- FDA approval represents a monumental milestone for patients with MPS IIIA and validates Abeona’s foundational R&D and clinical development efforts - - Ultragenyx successfully brought a life-changing therapy across the finish line for MPS IIIA patients and families - CLEVELAND, Sept. 18, 2026 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a commercial-stage biopharmaceutical company developing genetic medicines for serious diseases with high unmet need, today congratulated Ultrage

Ultragenyx prices Sanfilippo gene therapy at $4M; Bristol Myers shelves Orum drug

Ultragenyx’s second gene therapy approval since August gives it another sellable voucher. Elsewhere, Roche declared success in a blood cancer trial and a cell therapy maker is cutting costs.