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Neuromuscular Disease

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March 12, 2025
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FDA approves Soliris for patients aged 6 years and up with generalized myasthenia gravis

FDA approves Soliris for patients aged 6 years and up with generalized myasthenia gravis

The FDA has approved an expanded indication of Soliris, an IV-administered monoclonal antibody for patients aged 6 years and older with generalized myasthenia gravis who are anti-acetylcholine receptor antibody positive.

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July 15, 2024
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Prenatal air pollution exposure tied to increased risk for cerebral palsy

Prenatal air pollution exposure tied to increased risk for cerebral palsy

Prenatal exposure to ambient air pollution was associated with increased risk for children developing cerebral palsy, according to cohort study results published in JAMA Network Open.

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March 21, 2023
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Radicava ORS demonstrates long-term safety, tolerability in patients with ALS

Radicava ORS demonstrates long-term safety, tolerability in patients with ALS

Results from a phase 3 clinical trial showed that Radicava ORS was safe in and well-tolerated by patients with ALS after 48 weeks of treatment, according to a manufacturer press release.

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March 06, 2023
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FDA clears electrical muscle stimulator suit for neuromuscular injuries, disease

FDA clears electrical muscle stimulator suit for neuromuscular injuries, disease

The FDA has granted 510(k) clearance to Neuro20 Technologies for its Neuro20 Pro System, a wearable whole-body electrical muscle stimulator suit, software and operating system for neuromuscular injuries and disease.

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February 03, 2023
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Amylyx announces end of enrollment for phase 3 trial of ALS therapeutic

Amylyx announces end of enrollment for phase 3 trial of ALS therapeutic

Amylyx Pharmaceuticals Inc. announced completion of enrollment for a global, phase 3 clinical trial of AMX0035, an oral, fixed-dose medication for ALS.

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May 25, 2022
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TDP-43 accumulation likely indicator for patients with ALS

TDP-43 accumulation likely indicator for patients with ALS

Presence of axonal phosphorylated transactive response DNA-binding protein 43, or pTDP-43, accumulation in intramuscular nerve bundles is a likely biomarker for patients diagnosed with ALS, according to a study published in JAMA Neurology.

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February 03, 2022
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High mortality, health care use for patients with hypercapnic respiratory failure

High mortality, health care use for patients with hypercapnic respiratory failure

Hospitalized patients with compensated hypercapnic respiratory failure have high rates of mortality and health care utilization, with higher elevated carbon dioxide partial pressure associated with worse survival, researchers reported.

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October 26, 2021
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Among activity types, moderate exercise provides best ALS protection

Among activity types, moderate exercise provides best ALS protection

Lifetime physical activity did not appear to increase risk for ALS, whereas occupational physical activity seemed to be a risk factor for the disease, according to study results published in Neurology.

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November 13, 2020
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Vamorolone demonstrates efficacy, safety for boys with Duchenne muscular dystrophy

Vamorolone demonstrates efficacy, safety for boys with Duchenne muscular dystrophy

Treatment with vamorolone, an anti-inflammatory steroidal drug, improved motor outcomes in boys with Duchenne muscular dystrophy compared with corticosteroid-naive individuals, according to findings published in PLoS Medicine.

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September 03, 2019
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LMNA gene mutation linked to neuromuscular, cardiac conditions in most patients

Patients with LMNA gene mutations are likely to develop life-threatening neuromuscular or cardiac conditions, according to study results published in the Annals of Internal Medicine.