Diseases Targeted
The MDA targets the following muscular dystrophy diseases:
- Duchenne muscular dystrophy
- Becker's muscular dystrophy
- Emery-Dreifuss muscular dystrophy
- Limb-girdle muscular dystrophy
- Amyotrophic lateral sclerosis
- Congenital muscular dystrophy
It also targets the following:
- Infantile spinal muscular atrophy
- Juvenile, Intermediate, and Adult spinal muscular atrophy
- Spinal bulbar muscular atrophy
- Dermatomyositis
- Polymyositis
- Inclusion body myositis
- Myasthenia gravis
- Lambert-Eaton myasthenic syndrome
- Congenital myasthenic syndrome
- Hyperthyroid myopathy
- Hypothyroid myopathy
- Charcot-Marie-Tooth disease
- Friedreich's ataxia
- Dejerine-Sottas disease
- Myotonia congenita, both Thomsen's and Becker's Disease
- Paramyotonia congenita
- Central core disease
- Nemaline myopathy
- Myotubular myopathy (Centronuclear myopathy)
- Periodic paralysis, both Hypokalemic and Hyperkalemic
- Mitochondrial myopathy, a mitochondrial disease
- Facioscapulohumeral muscular dystrophy, FSHD
It also targets muscle diseases due to deficiencies in carnitine and the following enzymes:
- Phosphorylase
- Acid Maltase (Pompe's disease)
- Phosphofructokinase
- Debrancher enzyme (also known as Amylo-1,6-glucosidase); a glycogen storage disease also known as Forbes disease
- Carnitine palmityl transferase
- Phosphoglycerate kinase
- Phosphoglycerate mutase
- Lactate dehydrogenase
- Myoadenylate deaminase
Read more about this topic: Muscular Dystrophy Association
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