February 29, 2020
Article
The RareWear program is designed to bridge medical device companies to rare disease patient applicants, seeking better disease and treatment monitoring.
New research suggests proposals aimed at reducing out of pocket costs may harm minimally-funded drug developers.
November 08, 2018
A new, fundamental biological process in CLN8 disease, a form of Batten disease, has been discovered by a team of investigators from Baylor College of Medicine.
May 01, 2018
Lyso-Gb1 Identified as most efefctive biomarker for Gaucher disease monitoring.
April 07, 2018
Tarekegn Geberhiwot, MD, of the Institute of Metabolism and Systems Research and colleagues had an article published in the Orphanet Journal of Rare Diseases, outlining the development of clinical management recommendations for Niemann-Pick disease type C.
February 02, 2018
The director of the Ara Parseghian Medical Research Fund promoted the organization’s new initiatives to raise money for NPC1 research during an address to Notre Dame’s student senate this week.
January 19, 2018
Arimoclomol, in development by Danish biotech Orphazyme A/S for the treatment of Niemann-Pick Disease Type C, was granted rare pediatric disease designation by the U.S. FDA this morning.
