A study led by researchers at the National Institute of Neurological Disorders and Stroke (NINDS) recently published in the journal Neuron revealed new insights into why mutations in the SOD1 gene lead to the development of amyotrophic lateral sclerosis (ALS). The study is entitled “…
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Arrowhead Pharmaceuticals will seek permission later this year for the first clinical trial of ARO-SOD1, its RNA-based therapy for amyotrophic lateral sclerosis (ALS), according to a company press release. Further details about Arrowhead’s plans for testing its investigational RNA therapy are expected at a company Research and…
The greater incidence of amyotrophic lateral sclerosis (ALS) among professional athletes and people with head trauma, and the faster functional decline seen in animal models after localized nerve injury, strongly suggests that nerve injury plays a role in initiating disease processes, a review study reported. Needed avenues of future…
A single protein called Gemin3 may be the bridge linking the varied proteins known to underlie amyotrophic lateral sclerosis (ALS), suggesting a common pathway in this disease, a study using a fruit fly model showed. The discovery of this common…
A compound found in rice bran oil may lower oxidative stress levels and may be beneficial to people with amyotrophic lateral sclerosis, an early study in fruit flies and disease-related cell models shows. These findings were reported in the study “γ-Oryzanol mitigates oxidative stress and prevents mutant SOD1-Related neurotoxicity…
More than 50% of the cases of amyotrophic lateral sclerosis (ALS) disease are linked with a genetic cause, and first-degree relatives of an ALS member — especially daughters of ALS mothers — show the highest risk to inherit it, according to an Irish population-based study. The study “Lifetime Risk…
The Muscular Dystrophy Association (MDA) is financing eight new research projects aimed at developing new treatments and markers of disease progression for amyotrophic lateral sclerosis (ALS). The projects will investigate ALS mechanisms of disease, genetic causes, and pinpoint new therapy targets that can be used to develop better treatments. The…
Mutations in a gene linked to familial amyotrophic lateral sclerosis (ALS), called FUS, affect the ability of motor nerve cells to repair damage to their DNA, a new study shows. These findings suggest that therapies that enhance the repair of DNA damage may be of benefit to some ALS patients.
A gene therapy that might treat familial amyotrophic lateral sclerosis (ALS), an inherited form of the disease, was recently granted orphan drug status by the European Medicines Agency (EMA), an award that carries incentives to promote potential treatments for rare diseases. The investigative therapy is in preclinical development…
AveXis, which is running clinical trials of a promising gene therapy for spinal muscular atrophy (SMA) — and developing a potentially similar therapy for a genetic form of amyotrophic lateral sclerosis (ALS) — announced plans to be acquired by Novartis for $8.7 billion in cash. AVXS-101 is the…