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Rare Disease
Trials & Approvals Blood Cancer
FDA Approves First Interferon Therapy for Rare Blood Cancer
2021-11-15
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
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LATEST
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Study: Aligning U.S. Drug Prices with Europe Could Reduce American Life Expectancy by Six Months
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Tribal Leaders Warn Senators: Federal Health Funding Cuts Threaten Native American Health.
2025-05-14
Health Secretary Kennedy Retracts Parts of Agency Reorganization Plan During Combative Congressional Hearing
2025-05-14
SURMOUNT-5 Trial: Zepbound Shows Greater Weight Loss Than Wegovy
2025-05-14
ASCGT Meeting Navigates Biotech Downturn After Multi-Year Slump
2025-05-14
READ MORE
EVENT
2025-05-13
ASGCT 28th Annual Meeting 2025
New Orleans, U.S.A.
2025-05-26
2025 Antibody Therapeutic & Frontiers in Biomedical Sciences Conference
Taipei, Taiwan
2025-05-30
ASCO Annual Meeting 2025
Chicago, U.S.A
2025-06-11
ISSCR 2025 Annual Meeting
Hong Kong
2025-06-16
US BIO International Convention
Boston, U.S.A.
SEE MORE
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