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
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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
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NGF Mourns Loss of Rosina Papantonio
The NGF is profoundly saddened by the recent passing of... Read more
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Chart Your Own Course: A Scholarship Program for the LSD Community
What Is Chart Your Own Course? Chart Your Own Course... Read more
News
FDA Approves 23andMe Genetic Health Risk Reports
Earlier this month, 23andMe, the leading personal genetics company, announced... Read more
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Parkinson’s Progression Markers Initiative
Be Part of the Genetics Research Revolution People with Gaucher... Read more
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The Complicated Relationship between Gaucher Disease and Parkinsonism: Insights from a Rare Disease
Medical Advisory Board member, Dr. Ellen Sidransky, recently published an... Read more
News
Sanofi Initiates Phase 2 Clinical Trial to Evaluate Therapy for Genetic Form of Parkinson’s Disease
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Sanofi Genzyme, the specialty care global business unit... Read more
