News
AVROBIO to End Gaucher Disease Trials, Explore Sale
AVROBIO, a promising developer of clinical-stage gene therapy solutions, has announced... Read more
News
Gain Therapeutics, Inc. Announces Breakthrough Pre-Clinical Data of Novel Small Molecules for the Treatment of Parkinson’s Disease and Gaucher Disease
Study results demonstrate first small molecule approach to significantly reduce... Read more
Blog
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Living With Gaucher Disease
Committing to Care, Access, Equity, and Research: NORD Announces 31 Rare Disease Centers of Excellence
NORD provides new designation to medical centers to define standards... Read more
News
Gain Therapeutics, Inc. Announces Positive Topline Data in Patient-derived iPSC Study Evaluating STAR Compounds as a Treatment for Gaucher and Parkinson’s Disease
GT-02287and GT-02329 compounds display positive effects on GCase activity and... Read more
News
AVROBIO Reports Second Quarter 2021 Financial Results and Provides Business Update
U.S. Food and Drug Administration granted Fast Track Designation to... Read more
News
Gain Therapeutics Announces Issuance of Two New PCT Patents Covering GBA and GALC Modulator Compounds for Treatment of CNS Disorders
Activity of GBA and GALC Enzymes Play Key Roles in... Read more
News
Takeda Announces Creation of the Rare Disease Clinical Activity Protocols (Rare-CAP) program
A Five-year Commitment From Takeda Will Help Children’s National Launch... Read more
News
AVROBIO Receives Orphan-Drug Designation from FDA for AVR‑RD‑02
AVROBIO announced that the U.S. Food and Drug Administration (FDA)... Read more
News
Prevail Therapeutics Announces Investigational New Drug Trial for Parkinson’s Disease
U.S. Food and Drug Administration (FDA) has accepted Prevail’s Investigational... Read more
News
NGF Mourns Loss of Rosina Papantonio
The NGF is profoundly saddened by the recent passing of... Read more
