Listen
TRANSLARNA (ataluren) - Duchenne muscular dystrophy
Opinions on drugs -
Posted on
Nov 07 2024
Reason for request
Reassessment at the request of the CT
Summary of opinion
Unfavourable opinion for reimbursement in “the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years and older”.
Clinical Benefit
| Insufficient |
The clinical benefit of TRANSLARNA 125 mg, 250 mg, 1,000 mg (ataluren) granules for oral suspension is insufficient in the MA indication to justify public funding. |
Clinical Added Value
| Not applicable |
Documents
English version
Contact Us
Évaluation des médicaments
