Quote this publication Share Print

TRANSLARNA (ataluren), RNA interference agent

RARE DISEASE - NEW MEDICINAL PRODUCT
Opinions on drugs - Posted on Feb 23 2015

Reason for request

Inclusion

Minor improvement in the management of Duchenne muscular dystrophy

 

  • TRANSLARNA has Marketing Authorisation in the treatment of Duchenne muscular dystrophy (DMD) resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 5 years and older.
  • Available data suggest a potential benefit, poorly demonstrated and modest, in terms of slowing the loss of ambulation in some children with Duchenne muscular dystrophy, with acceptable safety.
  • Efficacy has not been demonstrated in patients who have lost ambulation.

Actual benefit

Modéré

-


Improvement in actual benefit

IV (mineur)

-


Therapeutic use

-

Contact Us

Évaluation des médicaments