MYOBASE

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MYOBASE THE BIBLIOGRAPHIC DATABASE ON NEUROMUSCULAR DISORDERS

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What is a neuromuscular disease?

A neuromuscular disease is a genetic, inflammatory or autoimmune, rare, progressive and severely disabling illness.
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ABOUT MYOBASE

Myobase is implemented by  AFM-Téléthon library which has been collecting documents on Neuromuscular Disorders since 1990.
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How to use Myobase ?

INSTITUTE OF MYOLOGY NEWSLETTERS


AFM-Téléthon honored as "Pioneer in Technology Development"

Los Angeles, March, 16, 2019 – AFM-Téléthon received the « Pioneer in Technology Development » award for its role in the development of innovative therapies for neuromuscular and rare diseases at the Annual World Congress of Society for Brain Mapping and Therapeutics (SBMT-Brain and Spine). Considered as the equivalent of the Oscar for Neuroscience, the … [Read more]

Cet article AFM-Téléthon honored as "Pioneer in Technology Development" est apparu en premier sur Institut de Myologie.


Corneal involvement of patients with polymyositis and dermatomyositis

A European team reports for the first time a certain tendency towards dry eye and thinning of the cornea in two inflammatory myopathies. To evaluate corneal parameters in patients with polymyositis (PM) and dermatomyositis (DM) and compare them with those of healthy controls, the authors enrolled a total of 43 PM and 32 DM patients … [Read more]

Cet article Corneal involvement of patients with polymyositis and dermatomyositis est apparu en premier sur Institut de Myologie.


I-Motion allowed to enroll type 1 SMA patients in the STR1VE trial developed by AveXis

The European gene therapy trial STR1VE EU is an open-label, phase III study to evaluate the efficacy and safety of utilisation of a single, intravenous injection of AVXS-101 (an AAV9 carrying the SMN1 gene), in 30 infants with SMA associated proximal spinal muscular atrophy (SMA) type 1, under 6 months of age. The study was … [Read more]

Cet article I-Motion allowed to enroll type 1 SMA patients in the STR1VE trial developed by AveXis est apparu en premier sur Institut de Myologie.


Actualités AFM-Téléthon


DMD : caractériser les troubles du développement neurocognitif
Maladies
Les résultats d'une étude d'histoire naturelle précisent la proportion des troubles neurocognitifs chez 204 garçons atteints de dystrophie musculaire de Duchenne.

Dystrophie musculaire de Duchenne : bonne tolérance de l'edasalonexent (CAT-1004)
Maladies
Un essai de phase I en ouvert montre la bonne tolérance et l'innocuité de l'edasalonexent (CAT-1004) oral chez de jeunes garçons atteints de DMD

Maladie de Pompe: prévention de l'immunité anti-GAA dans un modèle murin
Maladies
Une stratégie de thérapie génique permet de prévenir la réaction immunitaire anti-GAA et entraîne une expression durable de la protéine dans des souris déficientes en GAA