Muscular Dystrophy Association - Diseases Targeted

Diseases Targeted

The MDA targets the following muscular dystrophy diseases:

1. Duchenne muscular dystrophy
2. Becker's muscular dystrophy
3. Emery-Dreifuss muscular dystrophy
4. Limb-girdle muscular dystrophy
5. Amyotrophic lateral sclerosis
6. Congenital muscular dystrophy

It also targets the following:

1. Infantile spinal muscular atrophy
2. Juvenile, Intermediate, and Adult spinal muscular atrophy
3. Spinal bulbar muscular atrophy
4. Dermatomyositis
5. Polymyositis
6. Inclusion body myositis
7. Myasthenia gravis
8. Lambert-Eaton myasthenic syndrome
9. Congenital myasthenic syndrome
10. Hyperthyroid myopathy
11. Hypothyroid myopathy
12. Charcot-Marie-Tooth disease
13. Friedreich's ataxia
14. Dejerine-Sottas disease
15. Myotonia congenita, both Thomsen's and Becker's Disease
16. Paramyotonia congenita
17. Central core disease
18. Nemaline myopathy
19. Myotubular myopathy (Centronuclear myopathy)
20. Periodic paralysis, both Hypokalemic and Hyperkalemic
21. Mitochondrial myopathy, a mitochondrial disease
22. Facioscapulohumeral muscular dystrophy, FSHD

It also targets muscle diseases due to deficiencies in carnitine and the following enzymes:

1. Phosphorylase
2. Acid Maltase (Pompe's disease)
3. Phosphofructokinase
4. Debrancher enzyme (also known as Amylo-1,6-glucosidase); a glycogen storage disease also known as Forbes disease
5. Carnitine palmityl transferase
6. Phosphoglycerate kinase
7. Phosphoglycerate mutase
8. Lactate dehydrogenase
9. Myoadenylate deaminase