FAQ - Duchenne Muscular Dystrophy (DMD)
We have prepared an overview of the most frequently asked questions and provided answers which will hopefully clarify some of your questions. These answers have been prepared by specialists at Prosensa and our academic partner LUMC.
Duchenne Muscular Disorder FAQ
- My child has a mutation of “…..”, which exon should be skipped?
- Why is it important to know my (child's) mutation for exon skipping?
- What genetic test should be done to indicate if exon skipping could potentially help?
- Does exon skipping work for all mutations that disrupt the genetic code?
- Does exon skipping work for duplications?
- Does exon skipping also work for Becker patients?
- What will the clinical effect of exon skipping be?
- Can exon skipping restore muscle that is already lost?
- Can patients who are already in a wheelchair be treated with exon skipping?
- My child needs an exon “…“ skip, when will Prosensa start working on this?
- How can my child participate in an exon skipping clinical trial?
- Where can I find the latest information about clinical trials?
- Why do trials only involve a small number of patients? Wouldn’t it be fairer to include more children?
Still have a question?
If you have additional questions or if you would like further clarification, please send an email to Dr. Giles Campion, our Chief Medical Officer. He or one of his colleagues will try to answer your questions rapidly.
Dr. Giles Campion
Chief Medical Officer - SVP R&D - Prosensa
Languages: English
Timezone: GMT +1 hour (The Netherlands)
Phonework: +31 (0)71 3322100
E-mail: patientinfo@prosensa.nl
