Risdiplam
Risdiplam
Risdiplam (pronounced riz-DIP-lam) is a medication used in the treatment of Spinal Muscular Atrophy (SMA). It is an orally administered drug that increases the production of the Survival Motor Neuron (SMN) protein, which is deficient in individuals with SMA.
Etymology
The name "Risdiplam" is a proprietary name given by the drug's manufacturer, Roche. The exact etymology is not publicly disclosed, but pharmaceutical names often have roots in the drug's chemical structure, mechanism of action, or intended use.
Usage
Risdiplam is used to treat all types of Spinal Muscular Atrophy (SMA), a rare neuromuscular disorder. It is approved for use in patients 2 months of age and older. The drug works by modifying the SMN2 gene to increase production of the SMN protein.
Related Terms
- Spinal Muscular Atrophy (SMA): A genetic disorder characterized by weakness and wasting (atrophy) in muscles used for movement (skeletal muscles).
- Survival Motor Neuron (SMN) Protein: A protein that is crucial for the maintenance of motor neurons. Deficiency of this protein is the primary cause of SMA.
- Roche: The pharmaceutical company that manufactures Risdiplam.
- SMN2 gene: A gene that produces the SMN protein. Risdiplam modifies this gene to increase SMN protein production.
External links
- Medical encyclopedia article on Risdiplam
- Wikipedia's article - Risdiplam
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