Articles

Finance
May 16, 2022
Rare Disease Drug Developers Face Bear Market in April
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Finance
May 16, 2022
Kriya Raises $270 Million to Advance Fully Integrated Gene Therapy Engine
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Deals
May 16, 2022
KemPharm to Acquire Orphazyme, Expanding its Rare CNS Diseases Pipeline
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Drug Development
May 16, 2022
Rocket Reports Updates of Data on Multiple Gene Therapy Programs
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RARECast Podcasts
May 13, 2022
Targeting ALS with Synergistic Combinations of Therapies
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Deals
May 13, 2022
BridgeBio Sells Rare Pediatric Disease Priority Review Voucher for $110 Million, Defers Debt Payment
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Drug Development
May 13, 2022
FDA Approves Oral Form of Radicava for Treatment of Adults with ALS
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Drug Development
May 12, 2022
Caribou Reports Positive Initial Data for Gene-Edited Allogeneic CAR-T Cell Therapy
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Deals
May 12, 2022
BridgeBio Licenses SHP2 Inhibitor to Bristol Myers Squibb in Oncology
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Drug Development
May 12, 2022
Takeda Notifies Ovid of Delays in Pivotal Study of Therapy for Rare Epilepsies
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News

May 16, 2022
RARE Global Advocacy Alliance Member Spotlight: Ca...
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May 12, 2022
At a Crossroad for Innovation in Rare Disease
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May 5, 2022
Getting Your Community Ready for Clinical Trials
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May 5, 2022
What Does it Take to Become a Research-Ready Organ...
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May 2, 2022
Cómo puede ayudarle un asesor genético
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May 2, 2022
Pruebas clínicas para encontrar respuestas
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