Jun 12, 2018
E-Scape Bio Obtains Worldwide Rights from AbbVie for Selective S1P5 Program Targeting CNS-related Lysosomal Storage Disorders
Jun 09, 2018
Global Batten Community Unites To Launch Inaugural International Batten Disease (NCL) Awareness Day
May 01, 2018
Jill Abramson, Barbara Corcoran and Katie Couric to join National Tay-Sachs & Allied Diseases Association in New York City
Apr 24, 2018
New England Journal of Medicine Published Open-label Study Showing Brineura® (cerliponase alfa) Reduced the Rate of Clinical Decline of Children with CLN2 Disease, a Form of Batten Disease
Apr 09, 2018
Perlara announces partnership with Multiple Sulfatase Deficiency Action Foundation
Feb 26, 2018
Polaryx Therapeutics Received Orphan Drug Designation From the European Medicines Agency (EMA) for the Treatment of Neuronal Ceroid Lipofuscinosis With PLX-200
Feb 06, 2018
WORLDSymposium Recognizes BioMarin's Brineura® (cerliponase alfa) with 2018 New Treatment Award
Dec 11, 2017
Polaryx Therapeutics Receives Orphan Drug Designation From the US FDA for the Treatment of Neuronal Ceroid Lipofuscinoses With PLX-100
Oct 30, 2017
Tampa Bay Cyclist Will Ride Coast to Coast to Find a Cure for Niemann-Pick Type C
Oct 18, 2017
Popular Science Names BioMarin's Brineura® (cerliponase alfa) One of the Top Health Innovations of 2017 with "Best of What's New" Award
Oct 18, 2017
When Life Gives You Lemons...Help Save Lives and Take the #NoPuckerChallenge To Raise Awareness for Niemann-Pick Type C
Oct 16, 2017
Enzyvant Initiates Farber Disease Natural History Study
Aug 14, 2017
Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Pipeline Review, H2 2017
Aug 07, 2017
Polaryx Therapeutics Receives Orphan Drug Designation From the U.S. FDA for PLX-200 for Rare Pediatric Disease
Aug 03, 2017
Lysosomal Storage Disorder Therapeutics Pipeline Analysis 2017
Jun 01, 2017
European Commission Approves Brineura(TM) (cerliponase alfa), the First Treatment for CLN2 Disease, a Form of Batten Disease and Ultra-Rare Brain Disorder in Children