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Rare Disease
Trials & Approvals Gene TherapyRare Disease
Orchard Therapeutics’ Gene Therapy for Rare Neurological Disorder Wins FDA Approval
2024-03-20
Partnerships Novo NordiskTargeted Protein Degradation
Neomorph Links Together With Novo Nordisk in a $1.46 Billion Molecular Glue Partnership Deal
2024-02-27
Funding Pompe diseaseRare Disease
Amicus Therapeutics Secures $430 Million Financing Pact with Blackstone to Propel Rare Disease Mission
2023-10-04
Trials & Approvals FDA Approval
FDA Approves First Treatment for Fibrodysplasia Ossificans Progressiva
2023-08-21
Trials & Approvals Duchenne Muscular Dystrophy-dmdOrphan Drug Designation
The U.S. FDA Grants Orphan Drug Designation to NS-089/NCNP-02 for the Treatment of DMD
2023-08-17
Trials & Approvals Rare Disease
Reata’s Groundbreaking Therapy for an Ultra-rare Neuromuscular Disease Secures FDA Approval
2023-03-02
Special biopharmam&a
GeneOnline’s Pick: Top 10 M&A Deals in 2022
2022-12-28
Trials & Approvals Rare Disease
AstraZeneca’s Treatment for Rare Throat Disease, Eosinophilic esophagitis, Fails Primary Endpoint
2022-10-25
Trials & Approvals Rare Disease
Bristol Myers Squibb’ sNDA for Rare Heart Disease Treatment Accepted by the FDA
2022-10-21
Funding FDA
Nineteen Grants and Two Contracts Grab $38 Million In Support From the FDA
2022-10-17
Trials & Approvals Albireo
Albireo Signals To Apply For FDA Approval After Trial Win
2022-10-12
Trials & Approvals AstraZeneca
AstraZeneca’s Rare Disease Treatment Wins Third Approval in European Union
2022-09-23
Trials & Approvals BridgeBio Pharma
BridgeBio and Sentynl Therapeutics’ Nulibry Gets Greenlighted In Europe
2022-09-21
Trials & Approvals Rare Disease
QOL Medical’s Sucraid Single-Use Containers Approved For Patients With Sucrase Deficiency
2022-09-07
Trials & Approvals Orphan Drug
BioCryst’s ALK-2 Inhibitor Granted FDA Orphan Drug Status For Rare Bone Disease
2022-09-01
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LATEST
Takeda, Astellas, and Sumitomo Mitsui Banking Declare Agreement For Early Drug Discovery Program Incubation in Joint Venture
2024-04-23
Ochre Bio Announces Partnership with Boehringer Ingelheim to Develop Novel Regenerative Treatments for Patients with Advanced Liver Disease
2024-04-22
Earth Day Awareness: Hospitals Embrace Sustainability Efforts
2024-04-22
WHO Raises Alarm: Bird Flu Threat to Humans an ‘Enormous Concern’
2024-04-19
The Legal Battlefield of Weight-Loss Drugs: Eli Lilly and Novo Nordisk on the Defensive
2024-04-18
Pharmacogenomics in Asia-Pacific: Nalagenetics CEO Levana Sani Offers Insights and Strategies
2024-04-17
Rice University Engineers Develop Miniature Brain Stimulator for Safer and Less-invasive Neuromodulation
2024-04-17
READ MORE
EVENT
2024-04-27
2024 Biomedical Final Pitch Competition
Room DA1620, Dana Building, Dana-Farber Cancer Institute, 99 Jimmy Fund Way, Boston, MA 02115
SEE MORE
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