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Results For "rare-diseases"

123 News Found

Datavant partners with Indegene to power clinical trial recruitment
Digitisation | September 21, 2025

Datavant partners with Indegene to power clinical trial recruitment

Collaboration matches more eligible patients, including those with rare and complex diseases, to appropriate clinical trials


FDA approves Sanofi’s BTK inhibitor for immune thrombocytopenia
Drug Approval | September 02, 2025

FDA approves Sanofi’s BTK inhibitor for immune thrombocytopenia

ITP is a disease of complex immune dysregulation leading to low platelet counts, bleeding, and reduced quality of life


Sarepta advances siRNA collaboration and sale of arrowhead equity investment
News | August 19, 2025

Sarepta advances siRNA collaboration and sale of arrowhead equity investment

Sarepta has also entered into an agreement with Arrowhead to transfer 2,660,989 shares of Arrowhead common stock in satisfaction of $50 million of the company’s previously announced $100 million milestone payment obligation


ImCheck’s ICT01 receives FDA ODD for treatment of acute myeloid leukemia
Clinical Trials | July 21, 2025

ImCheck’s ICT01 receives FDA ODD for treatment of acute myeloid leukemia

Clinical data showing unprecedented remission rates in newly diagnosed AML patients support advancing ICT01 into pivotal trials


Capricor receives FDA’s CRL for Deramiocel BLA for duchenne muscular dystrophy
News | July 15, 2025

Capricor receives FDA’s CRL for Deramiocel BLA for duchenne muscular dystrophy

Capricor’s BLA for Deramiocel received Priority Review in March 2025


Merck to acquire US Biopharma firm SpringWorks Therapeutics for enterprise value of $3.4 billion
Biopharma | April 28, 2025

Merck to acquire US Biopharma firm SpringWorks Therapeutics for enterprise value of $3.4 billion

Planned acquisition will strengthen the presence of Merck Healthcare in the United States and expand reach of SpringWorks’ therapeutic innovations to more patients with rare tumors worldwide


Vgenomics and Meril Genomics collaborate to revolutionise precision medicine
Biotech | April 22, 2025

Vgenomics and Meril Genomics collaborate to revolutionise precision medicine

Access to NIPT, TB and Rare Disease diagnostics to benefit thousands of patients nationwide


Budget 2025: Industry welcomes customs duty exemptions on 36 life-saving drugs and medicines
Policy | February 01, 2025

Budget 2025: Industry welcomes customs duty exemptions on 36 life-saving drugs and medicines

The Union Budget 2025-26 strengthens India's healthcare sector with an allocation of Rs. 99,858.56 crore, marking a 9.78% rise from Rs. 90,958.63 crore in 2024-25