• ABOUT MUSCULAR DYSTROPHY

    Not a disease but a genetic disorder that might occur even as a fresh mutation.

  • TYPES OF MUSCULAR DYSTROPHY

    Only organization in India that cares for all types of muscular dystrophy patients

  • Clinical Trials

    Facilitating access to clinical trials and research updates

  • DIAGNOSIS

    Are you aware of the type of your muscular dystrophy? Do the genetic test.

  • PREVENTION

    Don’t pass muscular dystrophy on to generations. You can stop it!

'Translarna’-the first ever drug for DMD patients from PTC Theraputics

The first ever drug for Duchenne Muscular Dystrophy patients with NON-SENSE mutation

About ‘Translarna’ (Ataluren) : ‘Translarna’ is the brand name of ‘Ataluren’ from PTC Therapeutics. It is a novel, orally administered small-molecule compound for the treatment of Duchene Muscular Dystrophy patients with a nonsense mutation. Ataluren was granted conditional marketing authorization in the European Union (Economic area) for the treatment of nmDMD in ambulatory patients aged five years and older. Translarna is the first treatment approved for the underlying cause of DMD.
Also, it is available in the US only to the US patients through clinical trials because it is still an investigational drug.

Will this drug be useful to all DMD patients?  NO.  ‘TRANSLARNA can be used ONLY by DMD patients with NONSENSE (Point) mutation.

Most common question by parents: I have done the ‘genetic test (MLPA) to my son already. The report has identified deletions/ mutations and confirmed it as DMD.  Can this drug be used by my son?

Answer: NO. MLPA test could not identify Nonsense/point mutations. Therefore, the drug cannot be used by your son without knowing whether or not he has the nonsense mutation.

How do I know whether my child has a nonsense mutation? : Gene sequencing by ‘Next Generation Sequencing (NGS) would help identify nonsense/point mutations.

Please contact info@mdfindia.org for further information.



Contact Us

Muscular Dystrophy Foundation India

Address : Thilagar Street, Thasildhar Nagar, Melamadai Road
Anna Nagar, Madurai-625 020

Land mark: Near Varasithi Vinayakar Temple
Bus stop: Suguna Store, Anna Nagar

Mobile:0999 43 40 121

Email : info@mdfindia.org