Press Release Hub logo
  • Submit a Release
  • Home
  • All
  • ESG
  • Health
  • Technology
  • Finance
  • Contributors

“One Month to Save Lives”: Families race against 1st August deadline to secure access to life-saving rare disease drug

Contributed by: PA Media Assignments

Logo

PA Media Assignments logo

Images

PA Media Assignments associated0
PA Media Assignments associated1
PA Media Assignments associated2

Tags

NPUK
Xenpozyme

More Like This

Sanofi responds to NICE negative FAD of olipudase alfa

Business Wire logo

Chiesi Global Rare Diseases Awards Research Grants to Advance Innovation in Lysosomal Diseases

PR Newswire associated0

Chiesi Global Rare Diseases Launches New Research Grant Initiative to Support Research Excellence in Lysosomal Storage Disorders

Azafaros Announces Initiation of two Global Phase 3 studies with Nizubaglustat in Niemann-Pick disease Type C (NPC) and GM1/GM2 gangliosidoses, respectively

Business Wire logo

Azafaros granted important regulatory designations and clearance by European authorities for global Phase 3 studies, to be initiated in 2025

New documentary highlights pioneering scientific research into new targets for treatment of motor neuron disease

Business Wire logo

AQNEURSA® (levacetylleucine) Recommended for EU Approval by the CHMP to Treat Niemann-Pick Disease Type C

PR Newswire associated0

Pierre Fabre Laboratories, committed to combating rare pediatric diseases via innovative therapies

Pages

  • Home
  • All
  • ESG
  • Health
  • Technology
  • Finance
  • Contributors

Press Release Hub

  • Our Services
  • Submit a Release

Websites

  • PA Media Group
  • PA Mediapoint
  • Distribution Packages

© PA Media Group. 2025

Terms of Use

Privacy Policy

Contact Us