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Policy Rare Disease
Big Pharma and Non-Profits Join NIH, FDA in New Rare Diseases Drive
2021-10-28
Trials & Approvals Gene Therapy
Sio Gene Therapies Touts Positive Safety and Biomarker Data for GM1 Gangliosidosis Candidate
2021-10-22
Trials & Approvals Rare Disease
FDA Greenlights First and Only Treatment for Cholestatic Pruritus with ALGS
2021-10-04
M&A Cardiovascular Disease
Merck Acquires Acceleron for $11.5 Billion, Gaining Access to a Phase 3 Cardiovascular Asset
2021-09-30
M&A Amyloidosis
AstraZeneca Acquires Caelum in $500m Deal to Strengthen Rare Diseases Division
2021-09-30
Trials & Approvals Gene Therapy
Cancer Fears Puts BioMarin’s Phearless Study on Hold
2021-09-07
Trials & Approvals FDA Approval
BeiGene’s Brukinsa Bags Second FDA Approval, to Rival Imbruvica in Treating a Rare Cancer
2021-09-06
Trials & Approvals Gene Therapy
Serious Adverse Event in Study Participant Marks Latest Roadblock for Astellas’ Gene Therapy Trial
2021-09-03
Trials & Approvals Rare Disease
Merck Fortifies Oncology Pipeline with FDA Win for HIF-2α Inhibitor, Welireg
2021-08-17
Technology Gene Therapy
New Gene Therapy Offers Hope to Reverse Rare Genetic Disorder in Children
2021-07-16
Trials & Approvals Kidney Disease
Venglustat’s Phase 2/3 Trial Failure Pushes Sanofi to Drop Kidney Disease Program
2021-06-02
Trials & Approvals Rare Disease
FDA Rejects Protalix’s Enzyme Replacement Therapy for Rare Genetic Disease
2021-04-29
Trials & Approvals Rare Disease
AnaptysBio’s Dermatology Drug Hits Roadblock in Phase 2 Trial
2021-03-09
Trials & Approvals Rare Disease
BridgeBio Bags First FDA Nod with Rare Disease Drug Acquired from Alexion
2021-03-03
R&D CRX-LCA
Researchers Test Gene Augmentation Therapy in Patient-Derived Retinal Organoids to Find Eye Disease Cure
2021-02-23
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