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Nemaline Myopathies (Nemaline Myopathy) Summary

Description: A group of inherited congenital myopathic conditions characterized clinically by weakness, hypotonia, and prominent hypoplasia of proximal muscles including the face. Muscle biopsy reveals large numbers of rod-shaped structures beneath the muscle fiber plasma membrane. This disorder is genetically heterogeneous and may occasionally present in adults. (Adams et al., Principles of Neurology, 6th ed, p1453)

Also Known As: Nemaline Myopathy; Rod Myopathy; Myopathies, Nemaline; Adult Onset Nemaline Myopathy; Autosomal Dominant Nemaline Myopathy Show All >>

Networked: 210 relevant articles (2 outcomes, 8 trials/studies) for this Disease

Key Drugs and Agents for Nemaline Myopathies

Efficacy Chart >>
Drugs and Important Biological Agents (IBA) related to treatments:
  1. Tyrosine (L-Tyrosine) : 1 outcome in 3 results : FDA 12
  2. Melphalan (Alkeran) : 1 outcome in 2 results : FDA 2 Generic
  3. Tropomyosin : 3 studies in 41 results : IBA
  4. Ryanodine Receptor Calcium Release Channel (Ryanodine Receptor) : 2 studies in 10 results : IBA
  5. nebulin : 1 study in 52 results : IBA
  6. Actins (F Actin) : 1 study in 22 results : IBA
  7. Proteins (Proteins, Gene) : 1 study in 17 results : IBA
  8. Myosins (Myosin) : 1 study in 8 results : IBA
  9. Muscle Proteins (Muscle Protein) : 1 study in 5 results : IBA
  10. Antibodies : 1 study in 2 results : IBA
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Diseases Related to Nemaline Myopathies

  1. Muscle Weakness
  2. Central Core Myopathy (Central Core Disease)
  3. Muscular Diseases (Myopathy)
  4. Congenital Structural Myopathies (Centronuclear Myopathy)
  5. Nemaline Myopathies (Nemaline Myopathy)
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Key Therapies for Nemaline Myopathies

Efficacy Chart >>
  1. Transplants (Transplant) : 1 outcome in 1 result
  2. Stem Cell Transplantation : 1 result
  3. Highly Active Antiretroviral Therapy (HAART) : 1 result
  4. Cardiopulmonary Resuscitation (CPR) : 1 result
  5. Organ Transplantation : 1 result
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