WebAtaluren is an oral protein restoration therapy for the treatment of nonsense mutation DMD. It acts by changing the way muscle cells interpret genetic information, coaxing them to produce a needed muscle protein called dystrophin despite the presence of a mutation in the dystrophin gene. The drug, which is taken by mouth, targets a particular ... WebSep 17, 2024 · Ataluren is an investigational treatment being developed to treat Duchenne muscular dystrophy ( DMD) resulting from a nonsense mutation in the dystrophin gene in …
NICE review does not recommend routine funding for Duchenne muscu…
Weba NICE HST of ataluren; or (ii) the expiry or termination of the MAA. The Guidance that NICE shall publish pursuant to clause 3.1 of the MAA shall take into account any data provided by the MAH about the Expanded Treatment Cohort in addition to the data provided pursuant to clause 3.1 of the MAA. WebOn 30 September 2024, NICE published its draft guidance on Translarna (also known as ataluren) .Translarna is used to treat Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene and is currently available through a Managed Access Agreement (MAA), which ends in January 2024. Duchenne muscular dystrophy affects … pro group incorporated
PTC Therapeutics Announces European Medicines Agency …
http://bo-rec2024.afm-telethon.fr/fr/fiches-maladies/dystrophie-musculaire-de-duchenne WebTranslarna (ataluren) EMA/423254/2024 Page 3/3 acknowledged that patients with Duchenne muscular dystrophy have an unmet need for treatment of this serious condition. Translarna has been given ‘conditional authorisation’. This means that there is more evidence to come about the medicine, which the company is required to provide. WebMar 27, 2024 · Ataluren preserved upper limb function in boys with nonsense mutation Duchenne muscular dystrophy, according to a poster presented at the MDA Clinical & Scientific Conference. kutztown university golden bear village west