Lysosomal Storage Disorders

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On the HCPLive Lysosomal storage disorders page, resources on the topics of medical news and expert insight into the rare diseases can be found. Content includes articles, interviews, videos, podcasts, and breaking news on lysosomal disorder disease research, treatment, and drug development.

Latest News

Rare Genomics Institute Launches Free Rare Disease Device Program
Rare Genomics Institute Launches Free Rare Disease Device Program

February 29th 2020

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
Medicare Part D Bills Could Harm Rare Disease Drug Development

February 29th 2020

New, Fundamental Biological Process Identified in Batten Disease
New, Fundamental Biological Process Identified in Batten Disease

November 8th 2018

Lyso-Gb1 Identified as Most Effective Biomarker for Gaucher Disease Monitoring
Lyso-Gb1 Identified as Most Effective Biomarker for Gaucher Disease Monitoring

May 1st 2018

Orphanet Publishes Clinical Management Guidelines for NPC
Orphanet Publishes Clinical Management Guidelines for NPC

April 7th 2018

More News

Foundation of Late Football Legend Leads the Way for NPC1 Research

Foundation of Late Football Legend Leads the Way for NPC1 Research

February 2nd 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.

Orphazyma A/S Granted Rare Pediatric Disease Designation for NPC Treatment

Orphazyma A/S Granted Rare Pediatric Disease Designation for NPC Treatment

January 19th 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.

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