Hope Through Research

At MDA, we take a big picture perspective across the full spectrum of neuromuscular diseases to uncover scientific and medical breakthroughs that accelerate treatments and cures. The power in our research approach is that we can often apply learnings from one disease to progress in others to bring urgently-needed answers to our families.

State of the Science

Research into treatments for neuromuscular diseases has progressed enormously in recent years. For many of our diseases, potential treatments are now under investigation either in laboratories or in the clinic. MDA-sponsored research has either directly or indirectly resulted in most of the potential treatments that are now in development. Click on any of the diseases below to find out about latest research into that disease, as well as to learn more about the potential treatments being studied.

Even studies that are not currently being funded by MDA are based on basic research into muscle and nerve biology, the causes of the diseases and basic technologies, that have been funded by MDA for decades. To find out more about how MDA sponsors research, go to our section about the Funding Process.

Be sure to visit What Has MDA Achieved? to learn more about MDA's research progress.


Acid Maltase Deficiency (AMD)

Amyotrophic Lateral Sclerosis (ALS)

Andersen-Tawil Syndrome


Becker Muscular Dystrophy (BMD)

Becker Myotonia Congenita

Bethlem Myopathy (LGMD)

Bulbospinal Muscular Atrophy (Spinal-Bulbar Muscular Atrophy)


Carnitine Deficiency

Carnitine Palmityl Transferase Deficiency (CPT Deficiency)

Central Core Disease (CCD)

Centronuclear Myopathy

Charcot-Marie-Tooth (CMT)

Congenital Muscular Dystrophy (CMD)

Congenital Myasthenic Syndromes (CMS)

Congenital Myotonic Dystrophy

Cori Disease (Debrancher Enzyme Deficiency)


Debrancher Enzyme Deficiency

Dejerine-Sottas Disease (DSD)

Dermatomyositis (DM)

Distal Muscular Dystrophy (DD)

Duchenne Muscular Dystrophy (DMD)

Dystrophia Myotonica (Myotonic Muscular Dystrophy)


Emery-Dreifuss Muscular Dystrophy (EDMD)

Endocrine Myopathies

Eulenberg Disease (Paramyotonia Congenita)


Facioscapulohumeral Muscular Dystrophy (FSH or FSHD)

Finnish (Tibial) Distal Myopathy

Forbes Disease (Debrancher Enzyme Deficiency)

Friedreich's Ataxia (FA)

Fukuyama Congenital Muscular Dystrophy (CMD)


Glycogenosis Type 10

Glycogenosis Type 11

Glycogenosis Type 2

Glycogenosis Type 3

Glycogenosis Type 5

Glycogenosis Type 7

Glycogenosis Type 9

Gowers-Laing Distal Myopathy


Hauptmann-Thanheuser MD (EDMD)

Hereditary Inclusion-Body Myositis

Hereditary Motor and Sensory Neuropathy (CMT)

Hyperthyroid Myopathy

Hypothyroid Myopathy


Inclusion-Body Myositis (IBM)

Inherited Myopathies

Integrin-Deficient Congenital Muscular Dystrophy


Kennedy Disease (SBMA)

Kugelberg-Welander Disease (SMA)


Lactate Dehydrogenase Deficiency

Lambert-Eaton Myasthenic Syndrome (LEMS)

Limb-Girdle Muscular Dystrophy (LGMD)

Lou Gehrig’s Disease (ALS)


McArdle Disease (Phosphorylase Deficiency)

Merosin-Deficient Congenital Muscular Dystrophy

Metabolic Diseases of Muscle

Mitochondrial Myopathy

Miyoshi Distal Myopathy

Motor Neurone Disease

Muscle-Eye-Brain Disease (CMD)

Myasthenia Gravis (MG)

Myoadenylate Deaminase Deficiency

Myofibrillar Myopathy (LGMD)

Myophosphorylase Deficiency

Myotonia Congenita (MC)

Myotonic Muscular Dystrophy (MMD)

Myotubular Myopathy (MTM or MM)


Nemaline Myopathy

Nonaka Distal Myopathy


Oculopharyngeal Muscular Dystrophy (OPMD)


Paramyotonia Congenita

Pearson Syndrome

Periodic Paralysis

Peroneal Muscular Atrophy (CMT)

Phosphofructokinase Deficiency

Phosphoglycerate Kinase Deficiency

Phosphoglycerate Mutase Deficiency

Phosphorylase Deficiency

Polymyositis (PM)

Pompe Disease (AMD)

Progressive External Ophthalmoplegia (PEO)


Rod Body Disease (Nemaline Myopathy)


Spinal-Bulbar Muscular Atrophy (SBMA)

Spinal Muscular Atrophy (SMA)


Tarui Disease (Phosphofructokinase Deficiency)

Thomsen Disease (Myotonia Congenita)


Ullrich Congenital Muscular Dystrophy


Walker-Warburg Syndrome (CMD)

Welander Distal Myopathy

Werdnig-Hoffmann Disease (SMA)


ZASP-Related Myopathy (LGMD)

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