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Rare Disease
Amicus Therapeutics Secures $430 Million Financing Pact with Blackstone to Propel Rare Disease Mission
2023-10-04
FDA Approves First Treatment for Fibrodysplasia Ossificans Progressiva
2023-08-21
The U.S. FDA Grants Orphan Drug Designation to NS-089/NCNP-02 for the Treatment of DMD
2023-08-17
Reata’s Groundbreaking Therapy for an Ultra-rare Neuromuscular Disease Secures FDA Approval
2023-03-02
GeneOnline’s Pick: Top 10 M&A Deals in 2022
2022-12-28
AstraZeneca’s Treatment for Rare Throat Disease, Eosinophilic esophagitis, Fails Primary Endpoint
2022-10-25
Bristol Myers Squibb’ sNDA for Rare Heart Disease Treatment Accepted by the FDA
2022-10-21
Nineteen Grants and Two Contracts Grab $38 Million In Support From the FDA
2022-10-17
Albireo Signals To Apply For FDA Approval After Trial Win
2022-10-12
AstraZeneca’s Rare Disease Treatment Wins Third Approval in European Union
2022-09-23
BridgeBio and Sentynl Therapeutics’ Nulibry Gets Greenlighted In Europe
2022-09-21
QOL Medical’s Sucraid Single-Use Containers Approved For Patients With Sucrase Deficiency
2022-09-07
BioCryst’s ALK-2 Inhibitor Granted FDA Orphan Drug Status For Rare Bone Disease
2022-09-01
Sanofi’s Rare Disease Drug Granted First-Ever FDA Approval for the Condition
2022-09-01
Everest, Calliditas Win First Approval for Rare Kidney Disease Drug in Europe
2022-07-18
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2023-11-30
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