Need to include ASMD as a notified condition in the National Policy for rare diseases

ASMD is highly variable and the age of onset, specific symptoms and severity of the disorder can vary dramatically from one person to another

Takeda to strengthen health system for rare diseases in India

These initiatives aim to improve access to healthcare for rare disease patients

Sanofi reaffirms commitment to rare diseases in India

Receives Subject Expert Committee recommendation to bring new therapies of Pompe, ASMD to India

Dr Sheela Nampoothiri appointed as co-chair of ICMR's Rare Diseases expert committee

Dr Ratna Puri from Sir Ganga Ram Hospital, Delhi is the Chairperson of the expert committee.

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

Sanofi’s GPRC5D monoclonal antibody earns ODD in US for multiple myeloma

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

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

Suraksha Diagnostic unveils Eastern India's largest genomics lab

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