Titre : | Advances in Duchenne muscular dystrophy and Becker muscular dystrophy |
Revue : | Avancées de la recherche |
Auteurs : | Myoinfo, Auteur ; Lorain S, Validateur |
Type de document : | Publication AFM |
Editeur : | AFM-TELETHON, 06/2023 |
Collection : | Savoir & Comprendre |
Pages : | 52 p |
Langues: | Anglais |
Mots-clés : | avancée de la recherche ; base de données ; dépistage néonatal ; description de la maladie ; dystrophie musculaire de Becker ; dystrophie musculaire de Duchenne ; dystrophinopathie ; essai clinique ; étude observationnelle ; physiopathologie ; prise en charge thérapeutique ; registre de malades ; saut d'exon ; thérapie cellulaire ; thérapie génique ; translecture des codons stop |
Résumé : |
Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) are rare genetic diseases which affect skeletal and cardiac muscle. They primarily occur in males but can also occasionally affect females. DMD manifests as progressive muscle weakness which begins during childhood, while BMD manifests as less severe muscle weakness which appears during childhood or adolescence, or even later in life.
This document, published to coincide with the AFM-Téléthon General Meeting 2023, presents DMD and BMD research news from the past year (treatments, a selection of ongoing studies and clinical trials, scientific and medical publications, etc.) > Duchenne muscular dystrophy (DMD) > Becker muscular dystrophy (BMD) |
Documents numériques (1)
![]() AV23_BDMD_78362_ENG_20230901.pdf Adobe Acrobat PDF |