May 17, 2019
NIH administers first clinical trial treatment for GM1 created by Auburn, UMass
May 02, 2019
CytRx Corporation Highlights Arimoclomol Clinical Milestone Guidance Provided by Licensee Orphazyme A/S
May 01, 2019
LAL-D Treatment Market Size Worth $954 Million by 2025 : Grand View Research, Inc.
Mar 21, 2019
Enzyvant's Investigational Farber Disease Enzyme Replacement Therapy, RVT-801, Receives FDA Fast Track and Rare Pediatric Disease Designations
Feb 28, 2019
Auburn University senior, who lost her brother to a rare genetic disease, is on a mission for a cure
Feb 20, 2019
Sangamo Announces FDA Acceptance of IND Application for ST-920 Gene Therapy Candidate for Fabry Disease
Feb 14, 2019
Global Fabry Disease Market Insights, Epidemiology and Market Report 2019
Feb 07, 2019
BioMarin Announces Ongoing Study Demonstrates Durable Treatment Benefit from Brineura® (cerliponase alfa) for 3 Years
Feb 04, 2019
Enzyvant to Highlight Investigational RVT-801 Program and Farber Disease Natural History Study at Upcoming WORLDSymposium(TM)
Nov 14, 2018
FDA Grants Orphan Drug Designation To RGX-181 Gene Therapy For The Treatment Of CLN2 Form Of Batten Disease
Nov 05, 2018
Global Niemann-Pick Disease Market and Competitive Landscape 2014-2023
Nov 05, 2018
REGENXBIO and Abeona Therapeutics Announce Worldwide Exclusive Licenses for the Treatment of Four Rare Lysosomal Storage Disorders Using NAV AAV9 Vector
Oct 26, 2018
Global Fabry Disease Market and Competitive Landscape Report 2018 - Drug Sales Forecast to 2023
Oct 15, 2018
Shining a Light on Diagnosis for Rare Genetic Disease Patients
Sep 13, 2018
CytRx Corporation Announces Receipt of Milestone Payment from Orphazyme A/S
Aug 30, 2018
REGENXBIO Announces Pipeline Expansion with RGX-181 Gene Therapy for CLN2 Form of Batten Disease
Aug 10, 2018
FDA approves new treatment for a rare genetic disorder, Fabry disease
Aug 10, 2018
Tay-Sachs Disease Forecast In 19 Major Markets 2018-2028: Risk Factors, Disease Diagnosis and Prognosis Along with Specific Variations by Geography and Ethnicity
Jul 19, 2018
Enzyvant Announces Collaboration with PerkinElmer to Enable Diagnostic Testing for Farber Disease
Jul 16, 2018
Fabry Disease Markets 2018-2028 by Gender and 5-Year Age Cohort