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.
Clinical Benefit
Moderate |
- |
Clinical Added Value
minor |
- |
Therapeutic use
- |
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