Product Catalog [{"id":"378","name":"Viltepso","price":1298.31,"sku":"Viltolarsen (viltolarsen)","brand":"NS Pharma","position":1,"category":"Diseases\/Neurology\/Duchenne Muscular Dystrophy"},{"id":"377","name":"Vyondys 53","price":277200,"sku":"Vyondys 53 (golodirsen)","brand":"Sarepta Therapeutics","position":2,"category":"Diseases\/Neurology\/Duchenne Muscular Dystrophy"},{"id":"349","name":"Viltolarsen","price":1298.31,"sku":"Viltolarsen (NS-065\/NCNP-01)","brand":"Nippon Shinyaku","position":3,"category":"Diseases\/Neurology\/Duchenne Muscular Dystrophy"},{"id":"318","name":"Exondys 51","price":0,"sku":"Exondys 51 (eteplirsen)","brand":"Sarepta Therapeutics, Inc.","position":4,"category":"Diseases\/Neurology\/Duchenne Muscular Dystrophy"},{"id":"46","name":"Calcort","price":13.6,"sku":"Calcort (deflazacort)","brand":"Merrell Dow\/Henning","position":5,"category":"Diseases\/Neurology\/Duchenne Muscular Dystrophy"}]

Duchenne Muscular Dystrophy

About duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is a genetic neuromuscular disorder characterised by progressive muscle weakness and wasting1. It is caused by mutations in the dystrophin gene on the X-chromosome which result in an absence of the dystrophin protein1, a protein that helps keep muscle cells intact2

It is the most prevalent neuromuscular disorder, affecting up to 1 out of 3,600 male births worldwide1. Symptoms usually appear during in early childhood, between the ages 3 and 5. The disease primarily affects boys, but can also affect girls as well2, although this is quite rare. The standard therapy that can delay DMD progression is based on corticosteroids. The two mainly used in DMD are deflazacort and prednisone/prednisolone1,3

References 

1. Duchenne Muscular Dystrophy: From Diagnosis to Therapy
    Falzarano, M.S. et al., Molecules, 2015.

2. Duchenne Muscular Dystrophy (DMD)
    MDA, cited on 13/03/2018.

3. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management
    Bushby, K. et al.,The Lancet, 2010.

  1. Viltepso
    Viltepso (viltolarsen)
    Duchenne Muscular Dystrophy
    €1,298.31
  2. Vyondys 53
    Vyondys 53 (golodirsen)
    Duchenne Muscular Dystrophy
    €277,200.00
  3. Viltolarsen
    Viltolarsen (NS-065/NCNP-01)
    Duchenne Muscular Dystrophy
    €1,298.31
  4. Exondys 51
    Exondys 51 (eteplirsen)
    Duchenne Muscular Dystrophy
  5. Calcort
    Calcort (deflazacort)
    Duchenne Muscular Dystrophy
    €13.60
View as Grid List

5 Items

per page
Set Descending Direction