News

UX111 offers cognitive, motor skill gains in Sanfilippo syndrome type A

Ultragenyx Pharmaceutical’s experimental gene therapy UX111 slows or halts the brain damage that affects communication and cognitive and motor skills in children with Sanfilippo syndrome type A, according to new trial data. While younger children receiving the one-time therapy saw gains in these functional domains compared with untreated age-matched…

Japan agency clears JR-441 trial for Sanfilippo syndrome type A

JCR Pharmaceuticals said it received permission from the Pharmaceuticals and Medical Devices Agency (PMDA), a regulatory agency in Japan, to start a Phase 1 clinical trial of JR-441, an experimental enzyme replacement therapy for Sanfilippo syndrome type A. The company didn’t provide details. “We are pleased that the…

Denali’s DNL126 selected by FDA for START pilot program

The U.S. Food and Drug Administration (FDA) has selected DNL126, an enzyme replacement therapy being developed by Denali Therapeutics for Sanfilippo syndrome type A, to join its START pilot program — fully, Support for Clinical Trials Advancing Rare Disease Therapeutics. As part of this pilot program, launched last…