Branaplam
Branaplam (pronounced: bran-a-plam) is a small molecule RNA splicing modulator developed by Novartis for the treatment of Spinal Muscular Atrophy (SMA).
Etymology
The name "Branaplam" is a coined term, with no known etymology.
Pharmacology
Branaplam works by increasing the production of the Survival Motor Neuron (SMN) protein, which is deficient in individuals with SMA. It modulates the splicing of the SMN2 gene, leading to an increase in functional SMN protein.
Clinical Trials
Branaplam has been studied in a Phase 1/2 clinical trial for the treatment of SMA. The trial demonstrated that Branaplam could increase SMN protein levels and improve motor function in individuals with SMA.
Related Terms
- Spinal Muscular Atrophy: A genetic disorder characterized by weakness and wasting (atrophy) in muscles used for movement (skeletal muscles).
- Survival Motor Neuron: A protein that is necessary for the normal function of nerves that control muscle movement.
- RNA splicing: A process that removes introns—non-coding sections of an RNA transcript—and joins the remaining sections together.
- Novartis: A Swiss multinational pharmaceutical company based in Basel, Switzerland.
See Also
External links
- Medical encyclopedia article on Branaplam
- Wikipedia's article - Branaplam
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