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Results For "rare-diseases"

109 News Found

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


AstraZeneca plans US$ 570 million investment in Canada
News | January 27, 2025

AstraZeneca plans US$ 570 million investment in Canada

700 new scientific and high-skilled jobs to be created in Greater Toronto Area, investing to execute more than 210 AstraZeneca global clinical studies


Zydus announces USFDA Orphan Drug Designation to Usnoflast for treatment of ALS
Drug Approval | January 23, 2025

Zydus announces USFDA Orphan Drug Designation to Usnoflast for treatment of ALS

The company is committed to unlocking new frontiers in neuroscience and developing Usnoflast for patients with ALS


Bayer’s pharma growth strategy progressing well as pipeline advances
News | January 15, 2025

Bayer’s pharma growth strategy progressing well as pipeline advances

Regulatory filings underway for a third indication for darolutamide in prostate cancer, for finerenone in a common form of heart failure, and acoramidis in transthyretin amyloid


FDA grants orphan drug designation to MicuRx's MRX-5 for non-tuberculous mycobacteria infections
Drug Approval | January 02, 2025

FDA grants orphan drug designation to MicuRx's MRX-5 for non-tuberculous mycobacteria infections

MRX-5 is a novel benzoxazole antibiotic developed for the treatment of mycobacteria infections, particularly infections caused by non-tuberculous Mycobacteria