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Rare Disease
Orchard Therapeutics’ Gene Therapy for Rare Neurological Disorder Wins FDA Approval
2024-03-20
Neomorph Links Together With Novo Nordisk in a $1.46 Billion Molecular Glue Partnership Deal
2024-02-27
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
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LATEST
Preventive Medicine for Neurodegeneration on the Rise: New Breakthrough in Routine Blood Testing for Alzheimer’s Disease
2024-05-20
Discrimination Leads to Rapid Aging, Science Shows the Detrimental Effects of this Act
2024-05-17
A New Study has Linked Treatment Resistant Depression to Body Mass Index
2024-05-16
AI Reveals Sex-Related Brain Differences and Precision Medicine Potential
2024-05-16
The EU Sustainability Directive’s Influence on Pharmaceutical Supply Chains
2024-05-15
New UC Berkeley Study Challenges Traditional Views on Lactate and Carbohydrate Metabolism
2024-05-15
First Person to Receive Transplanted Pig Kidney has Died
2024-05-13
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