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
Nov 1 ALNY Alnylam Q3 Loss In Line With Estimates, Revenues Miss, Stock Down
Nov 1 TVTX Travere Therapeutics Third Quarter 2024 Earnings: Revenues Beat Expectations, EPS Lags
Nov 1 BIIB Biogen partner Eisai completes FDA submissions for injectable Alzheimer’s therapy
Nov 1 ALNY Alnylam Pharmaceuticals Third Quarter 2024 Earnings: EPS Beats Expectations, Revenues Lag
Nov 1 PRAX Praxis Precision Medicines to Announce Third Quarter 2024 Financial Results and Host Conference Call on Wednesday, November 6, 2024
Nov 1 BIIB Biogen Third Quarter 2024 Earnings: Revenues Beat Expectations, EPS Lags
Nov 1 PTCT Regeneron battles Wall Street uncertainty; Madrigal extends fast launch of MASH drug
Nov 1 ALNY Alnylam Pharmaceuticals Inc (ALNY) Q3 2024 Earnings Call Highlights: Strong Revenue Growth and ...
Nov 1 ALNY Decoding Alnylam Pharmaceuticals Inc (ALNY): A Strategic SWOT Insight
Nov 1 ALNY Q3 2024 Alnylam Pharmaceuticals Inc Earnings Call
Oct 31 BIIB Biogen Q3 Earnings Review: Why I Remain Optimistic Despite Difficult Quarter
Oct 31 TVTX Travere Therapeutics, Inc. (TVTX) Q3 2024 Earnings Call Transcript
Oct 31 ALNY Alnylam Pharmaceuticals, Inc. 2024 Q3 - Results - Earnings Call Presentation
Oct 31 ALNY Alnylam Pharmaceuticals, Inc. (ALNY) Q3 2024 Earnings Call Transcript
Oct 31 BIIB Biogen down as Morgan Stanley cuts rating on sluggish Alzheimer’s drug launch
Oct 31 BIIB Biogen slips as Morgan Stanley downgrades on Leqembi launch
Oct 31 IONS Karyopharm Therapeutics (KPTI) May Report Negative Earnings: Know the Trend Ahead of Q3 Release
Oct 31 ALNY Alnylam (ALNY) Reports Q3 Earnings: What Key Metrics Have to Say
Oct 31 ALNY Alnylam Pharmaceuticals (ALNY) Reports Q3 Loss, Lags Revenue Estimates
Oct 31 ALNY Alnylam: Q3 Earnings Snapshot
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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