Spinal Muscular Atrophy Stocks List
Symbol | Grade | Name | % Change | |
---|---|---|---|---|
PTCT | B | PTC Therapeutics, Inc. | 0.53 | |
TVTX | C | Travere Therapeutics, Inc. | 3.02 | |
PRAX | C | Praxis Precision Medicines, Inc. | -0.17 | |
ALNY | D | Alnylam Pharmaceuticals, Inc. | 0.23 | |
IONS | F | Ionis Pharmaceuticals, Inc. | -1.07 | |
BIIB | F | Biogen Idec Inc. | 1.21 |
Related Industries: Biotechnology Drug Manufacturers - Major
Symbol | Grade | Name | Weight | |
---|---|---|---|---|
MNTL | D | Tema Neuroscience and Mental Health ETF | 11.93 | |
MEDX | F | Horizon Kinetics Medical ETF | 10.31 | |
BBP | C | BioShares Biotechnology Products Fund | 9.81 | |
BIS | B | ProShares UltraShort Nasdaq Biotechnology | 9.14 | |
PBE | D | PowerShares Dynamic Biotech & Genome | 8.99 |
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- Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is a rare neuromuscular disorder characterised by loss of lower motor neurons and progressive muscle wasting, often leading to early death.
The disorder is caused by a genetic defect in the SMN1 gene, which encodes SMN, a protein widely expressed in all eukaryotic cells (that is, cells with nuclei, including human cells) and necessary for survival of motor neurons. Lower levels of the protein results in loss of function of neuronal cells in the anterior horn of the spinal cord and subsequent system-wide atrophy of skeletal muscles.
Spinal muscular atrophy manifests in various degrees of severity, which all have in common progressive muscle wasting and mobility impairment. Proximal muscles, arm and leg muscles that are closer to the torso and respiratory muscles are affected first. Other body systems may be affected as well, particularly in early-onset forms of the disorder. SMA is the most common genetic cause of infant death.
Spinal muscular atrophy is an inherited disorder and is passed on in an autosomal recessive manner. In December 2016, nusinersen (marketed as Spinraza) became the first approved drug to treat SMA while several other compounds remain in clinical trials.
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