As a neurodegenerative disease characterized by childhood onset dementia, Sanfilippo syndrome causes immense suffering in many ways, including pain, loss of speech, extreme agitation, and distress, ...

(Las Vegas, Nevada, USA) DelveInsight’s “Sanfilippo Syndrome Market Insights, Epidemiology, and Market Forecast-2032” report delivers an in-depth understanding of Sanfilippo Syndrome, historical and ...

As a neurodegenerative disease characterized by childhood onset dementia, Sanfilippo syndrome causes immense suffering in many ways, including pain, loss of speech, extreme agitation, and distress, ...

As parents watch their kids transition from infancy into childhood, it’s common to look for signs of progressing development. Are they sleeping through the night? Can they remember new words and use ...

COLUMBUS, Ohio, Dec. 18, 2023 /PRNewswire/ -- Andelyn Biosciences, Inc., a pioneering and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), announced ...

Sanfilippo syndrome is a fatal, childhood, neurodegenerative, disorder—with no approved treatment—that involves neuroinflammation among multiple pathologies. Characterized by childhood onset dementia, ...

(RTTNews) - Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company, on Wednesday reported encouraging data from its ongoing clinical program of UX111, a gene therapy for Sanfilippo ...

Sanfilippo syndrome, also known as mucopolysaccharidosis type III (MPS III), is considered an orphan disease, which classifies it for special considerations in drug development and policy. It is a ...

Nine global foundations collaborate to grant $13.85 million for the continued advancement of lead Sanfilippo gene therapy programs Company receives infusion of an additional $5 million from exercise ...

Spruce Biosciences, Inc. (Nasdaq: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for neurological disorders with significant unmet medical need ...