Duchenne and Becker muscular dystrophy: Treatment & Research. Part II.
Investigations Genetic testing will confirm the diagnosis of Duchenne and Becker and show the exact mutation in the gene. Often the first test is a blood test measuring CK (creatine kinase), which is
Duchenne and Becker muscular dystrophy: types & causes. Part I.
What is Duchenne- and Becker Muscular Dystrophy? Duchenne and Becker MD are both X-linked recessive disorders. Both diseases are caused by mutations in the dystrophin gene, which encodes the protein
VISION-DMD App for boys with Duchenne Muscular Dystrophy
The Vision-DMD project aims to advance and accelerate the clinical development of the orphan drug Vamorolone —also known as VBP15— for the treatment of Duchenne Muscular Dystrophy (DMD) in ambulant
How to build an infrastructure for translational research
On 11th December 2018, Rebecca Leary, EURO-NMD Project Manager and member of the TREAT-NMD Secretariat and Cathy Turner, DMD Liaison and TACT Coordinator at the John Walton Muscular Dystrophy Research
How to guarantee quality in translational research for neuromuscular diseases
It is not easy to go from basic drug research to clinical trials on humans, and for rare neuromuscular diseases the scenario is even harder. Besides, not always all the projects arrive to the human