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Category
OrthopedicPublished By
GetWellGo TeamUpdated on
15-Sep-2025Myopathies are a general category of muscle disorder that may either be inherited (genetic) or acquired (inflammatory, metabolic, toxic, or endocrine-related). There are various types and severity of symptoms that are treated depending on their underlying cause. Here’s a structured overview:
Determine the type of underlying
Goals of treatment
Inflammatory Myopathies
Metabolic Myopathies
Endocrine Myopathies
Correct underlying hormone imbalance:
Myopathy associated with Drugs or Toxins
Genetic Muscular Dystrophies / Inherited Myopathies
There is no cure as yet; treatment is concerned with:
Supportive Measures & Lifestyle Measures
Myopathies are diseases that affect the muscles primarily, and cause weakness and other systemic complications. The symptoms differ depending on the nature of the myopathy (inherited (genetic) or acquired (inflammatory, metabolic, endocrine or drug-induced)) but share some common features.
General Symptoms of the Myopathies
Inherited myopathies treatment
Muscular dystrophies, congenital myopathies, mitochondrial myopathies, metabolic myopathies, and channelopathies, all of which are inherited myopathies (also known as genetic myopathies).
Most inherited myopathies are not fully curable since they have genetic defects and therefore treatment aims at slowing down, managing complications and enhancing quality of life.
General Management of Inherited Myopathies
Muscular Dystrophies (e.g., Duchenne, Becker, Limb-Girdle)
Corticosteroids (Prednisone, Deflazacort) → retard muscle deterioration.
Gene-based therapies (emerging):
Congenital Myopathies (e.g., nemaline, centronuclear, myotubular)
Mitochondrial Myopathies
No definitive cure.
Supportive therapies:
Metabolic Myopathies (glycogen storage disorders, fatty acid oxidation disorders)
Dietary modifications:
Channelopathies (e.g. periodic paralysis, myotonia)
Metabolic myopathies are a cluster of hereditary muscle disorders that occur due to energy metabolism defects (an inability to breakdown glycogen, an inability to oxidize fatty acids, or a failure to produce energy in a mitochondrion). Since underlying enzyme defects are genetic, a full cure is often not possible - but treatment is adapted to the metabolic defect responsible to minimize symptoms and crisis.
Glycogen Storage Diseases (GSD)
In the case of Pompes disease Alglucosidase alfa (life changing therapy).
Physical therapy (aerobic exercise has the power to enhance the efficiency of mitochondria).
Supplements / Mitochondrial cocktails (benefit varies):
Autoimmune muscle diseases are inflammatory myopathies which include polymyositis (PM), dermatomyositis (DM), necrotizing autoimmune myopathy (NAM), and inclusion body myositis (IBM).
The treatment is primarily aimed at the immune suppression, preservation of muscle functioning and management of complications.
General Treatment Goals
Corticosteroids (Prednisone/Prednisolone)
Immunosuppressive/ Immunomodulatory Therapy
High / Biologic Therapies
Inclusion Body Myositis (IBM)
Dr. Sumeet Agrawal
Dr. Rajiva Gupta
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