Spinal Muscular Atrophy Stocks List

Related ETFs - A few ETFs which own one or more of the above listed Spinal Muscular Atrophy stocks.

Spinal Muscular Atrophy Stocks Recent News

Date Stock Title
Mar 28 PRAX Biotech Stock Roundup: PRAX, VKTX Up on Study Results & MRNA, REGN Give Updates
Mar 28 PTCT PTC Announces Submission of Sepiapterin MAA for Treatment of PKU to EMA
Mar 28 BIIB Race To Treat Alzheimer's Disease Is Heating Up Despite Eli Lilly's Setback
Mar 28 IONS Ionis to hold olezarsen Phase 3 data webcast
Mar 28 PRAX Praxis Precision Medicines, Inc. Announces Pricing of $200 Million Public Offering
Mar 27 PRAX Praxis Precision Medicines announces proposed public offering
Mar 27 PRAX Praxis (PRAX) Surges on Positive Epilepsy Study Results
Mar 26 PRAX Praxis: 2nd Half 2024 ET Readout Could Lead To U.S. Regulatory Filing
Mar 26 BIIB CHMP to Delay Nod on Biogen (BIIB), Eisai's Alzheimer's Drug
Mar 26 PRAX Tesla, Krispy Kreme, McCormick And Other Big Stocks Moving Higher On Tuesday
Mar 26 PRAX Praxis, A Top 1% Stock, Soars To Year-High After Epilepsy Treatment Scores Big
Mar 26 PRAX Praxis gains after mid-stage data for epilepsy candidate
Mar 26 PRAX Praxis Precision Medicines Reports Positive Results of PRAX-628 Study Evaluating Photo Paroxysmal Response (PPR) Achieving 100% Response in Treated Patients
Mar 25 BIIB Biogen Inc. (BIIB) Dips More Than Broader Market: What You Should Know
Mar 25 PRAX Praxis Precision Medicines to Host PRAX-628 Program Update
Mar 25 PTCT A Comprehensive Look at Sarepta's Growth and Innovation
Mar 25 IONS Can Ionis' (IONS) Wholly-Owned Drugs Cut Dependency on Collabs?
Mar 25 IONS Positive olezarsen Phase 3 data in familial chylomicronemia syndrome to be presented at 2024 American College of Cardiology (ACC) annual meeting
Mar 25 BIIB Labcorp (LH) Boost Diagnostics Suite With New Biomarker Test
Mar 25 PRAX A Look At The Intrinsic Value Of Praxis Precision Medicines, Inc. (NASDAQ:PRAX)
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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