January 23, 2020
Genentech’s Risdiplam Meets Primary Endpoint in Pivotal SMA Type 1 Trial in Infants
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January 22, 2020
Making the Value Proposition for Rare Disease Therapies
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January 22, 2020
FDA Approves First Treatment for Rare Thyroid Eye Disease
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January 21, 2020
Stealth Initiates Clinical Study of Second-Generation Mitochondrial Candidate in Neurodegenerative Diseases
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January 17, 2020
Driving N-of-1 Therapies for Ultra-Rare Disease Patients
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January 17, 2020
Creating the Means to an End
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January 16, 2020
Cyprium Receives Rare Pediatric Disease Designation for Menkes Therapy
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January 13, 2020
LogicBio Enters Collaboration with Takeda to Develop New Genome Editing Therapy
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January 13, 2020
Ultragenyx Reports Positive Topline Results from OTC Deficiency Gene Therapy Study
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January 10, 2020
Cutting the Time and Cost of Drug Discovery with MicroOrgans
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January 10, 2020
FDA Approves First Targeted Therapy to Treat Rare Mutation in Patients with GIST
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January 10, 2020
FDA Grants Aruvant Rare Pediatric Disease Designation for SCD Therapy
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