Phase 2/3 Adult and Phase 1/2 Pediatric Clinical Trial Update
Representatives of the National Niemann-Pick Disease Foundation (NNPDF), Niemann-Pick UK and the International Niemann-Pick Disease Alliance (INPDA) today participated in a teleconference with Genzyme, A Sanofi Company.
We were pleased to learn that plans for the Phase 2/3 Adult and Phase 1/2 Pediatric Clinical trial of rhASM for ASMD Niemann-Pick disease continue to progress. Discussions continue with the regulatory authorities, including the FDA and the EMA and we expect Genzyme to release a more detailed community update soon.
Please contact the NNPDF Central Offices should you have any questions at: email@example.com