PTC Therapeutics dedicated innovator company working on ground breaking science to address unmet needs of rare diseases. They have approved products for Duchenne muscular dystrophy and spinal muscular atrophy.
Ikris Pharma Network is authorized by PTC Therapeutics to provide access to their medicines to Indian patients through Named-Patient Import.
PTC Products
Translarna™ (ataluren)
Translarna: Translarna™ (ataluren)* has been approved in the European Union for ambulatory patients aged 2 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene.
Translarna is a medicine that is used to treat patients aged 2 years and older with Duchenne muscular dystrophy who are able to walk. Duchenne muscular dystrophy is a genetic disease that gradually causes weakness and loss of muscle function. Translarna is used in the small group of patients whose disease is caused by a specific genetic defect (called a ‘nonsense mutation’) in the dystrophin gene.
Duchenne muscular dystrophy is rare, and Translarna was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 27 May 2005.
For more details:
https://www.ema.europa.eu/en/medicines/human/EPAR/translarna
https://www.ema.europa.eu/en/documents/overview/translarna-epar-medicine-overview_en.pdf
*Translarna is an investigational new drug in the US
