Résultats de la recherche - 6 results
A retrospective, non-interventional cohort study to compare adults with SMA receiving risdiplamwith untreated patients: Study design.
Walter, M.; Marlène Jagut; Haberlová, J.; Hodgkinson, V.; Yiu, E.; Murphy, L.; TREAT-NMD Global Data systems Oversight Committee (TGDOC); TREAT-NMD Global Registry Network for SMA; Roe, T.; ...
International collaboration to improve knowledge on myotonic dystrophy type 2
myotonic dystrophy type 2 non-invasive ventilation pacemaker Registry Abstract: Background: The TREAT- NMD Global Registry Network is a global collaboration of neuromuscular disease registries, including ...
Belgian neuromuscular diseases registry (BNMDR): Annual report 2017
evaluating the importance of neuromuscular diseases and patient characteristics, to promote health services for patients with a neuromuscular disease, to provide information to public health authorities for ...
Early stages of building a rare disease registry, methods and 2010 data from the Belgian Neuromuscular Disease Registry (BNMDR).
Retrospective Studies Abstract: The Belgian Neuromuscular Disease Registry, commissioned in 2008, aims to collect data to improve knowledge on neuromuscular diseases and enhance quality health services for ...
The TREAT-NMD DMD Global Database: analysis of more than 7,000 Duchenne muscular dystrophy mutations
Databases Development Diagnostics disease Diseases Duchenne muscular dystrophy electronic Frequency Genetic Genetic Therapy global im Institute IS journal Medicine Muscular Dystrophies Muscular dystrophy ...
The TREAT-NMD Duchenne muscular dystrophy registries: conception, design, and utilization by industry and academia
of national and ultimately global patient registries has been central to the success of TREAT- NMD. For the DMD registries within TREAT- NMD, individual countries have chosen to collect patient ...