Zoekresultaten - 2 results
Rapport annuel- registre belge des maladies neuromusculaires 2015
atrophies. Those specific data are also analyzed in the present report. The procedure for entry of data was assessed during this data collection. 5% of the registered files were compared to the original ...
The TREAT-NMD Duchenne muscular dystrophy registries: conception, design, and utilization by industry and academia
twenties/thirties. DMD is a rare disease (prevalence <5/10,000). Even the largest countries do not have enough affected patients to rigorously assess novel therapies, unravel genetic complexities, and determine ...