Duchenne Muscular Dystrophy (DMD) is an X-linked recessive disorder caused by loss-of-function mutations in the DMD gene, which disrupts dystrophin protein production in skeletal and cardiac muscle. It is one of the most common and severe forms of muscular dystrophy in children, almost always affecting boys.

Burden in India: India is estimated to have 400,000–500,000 children living with DMD, against a global incidence of roughly 1 in 3,600 male births. Awareness, early genetic diagnosis, and access to advanced therapies remain significant challenges – cost, infrastructure, and lack of specialist centers are the biggest barriers cited by clinicians.

Regulatory pathway for any DMD trial run in India:

CDSCO / DCGI – apex authority granting clinical trial permission, test licenses, and import/export permits (via the Sugam portal).
Review Committee on Genetic Manipulation (RCGM), under the Dept. of Biotechnology – reviews preclinical safety, vector shedding studies, and gene-therapy-specific risks.
Subject Expert Committees (SEC)– evaluate trial design and risk-benefit before DCGI sign-off.
Institutional Ethics Committees (IEC) / Institutional Biosafety Committees (IBSC) – local oversight, informed consent, biosafety.
CTRI (Clinical Trials Registry – India), hosted by ICMR – registration here is legally mandatory before enrollment starts. Always check a trial’s CTRI number before trusting it.

References:

Status of Clinical Care of DMD: Global Perspective and Situation in India, Indian Journal of Pediatrics (2025), https://link.springer.com/article/10.1007/s12098-025-05975-z
National Guidelines for Gene Therapy Product Development and Clinical Trials, ICMR, https://www.icmr.gov.in/icmrobject/custom_data/pdf/resource-guidelines/guidelines_GTP.pdf
CTRI, https://ctri.nic.in