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Lysosomal Storage Disorders
Rare Genomics Institute Launches Free Rare Disease Device Program
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.
Medicare Part D Bills Could Harm Rare Disease Drug Development
February 29, 2020
Article
New research suggests proposals aimed at reducing out of pocket costs may harm minimally-funded drug developers.
New, Fundamental Biological Process Identified in Batten Disease
November 08, 2018
Article
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.
Lyso-Gb1 Identified as Most Effective Biomarker for Gaucher Disease Monitoring
May 01, 2018
Article
Lyso-Gb1 Identified as most efefctive biomarker for Gaucher disease monitoring.
Orphanet Publishes Clinical Management Guidelines for NPC
April 07, 2018
Article
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.
Foundation of Late Football Legend Leads the Way for NPC1 Research
February 02, 2018
Article
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.
Orphazyma A/S Granted Rare Pediatric Disease Designation for NPC Treatment
January 19, 2018
Article
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.