Changes to a critical structure within the cell nucleus of spinal nerve cells — the nucleolus — preceded the appearance of other disease markers in people with either familial or sporadic forms of amyotrophic lateral sclerosis (ALS), a study has discovered. …
Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, blue, and purple lights side-by-side? These are the colors most often used to represent Rare Disease Day. This…
Under an agreement with the University of Kansas, PharmaTher has gained exclusive worldwide rights to develop and possibly commercialize ketamine as a therapy for amyotrophic lateral sclerosis (ALS). “We are pleased to have added the [ALS] program to our already impressive development pipeline…
Injecting a specific type of stem cells into the bloodstream was able to reduce muscle shrinkage and promote motor neuron survival in a mouse model of amyotrophic lateral sclerosis (ALS), a study found. According to investigators, the findings indicate these stem cells, known as multi-lineage differentiating stress enduring (Muse)…
The small difference in treatment responses among amyotrophic lateral sclerosis (ALS) patients receiving NurOwn versus a placebo in a recent clinical trial likely was due to chance, the U.S. Food and Drug Administration (FDA) announced in a press release. Along with slightly more deaths in the NurOwn…
PrimeC is safe and well-tolerated for the treatment of amyotrophic lateral sclerosis (ALS) and showed signs of slowing disease progression in patients, findings from a recently-completed Phase 2a study show. All 15 participants in the NST002 clinical trial (NCT04165850) have chosen to…
For the first time, a compound was able to restore the health of diseased upper motor neurons — nerve cells that carry voluntary movement signals from the brain to the spinal cord — in the brain of amyotrophic lateral sclerosis (ALS) mouse models, a study…
ALS Focus has opened a new survey looking specifically at the needs and challenges of caregiving for people with amyotrophic lateral sclerosis (ALS). Survey responses are expected to influence ALS programs and policy decisions. Register here to participate if you are an adult caregiver and live in the…
A novel system for three-dimensional (3D) imaging of zebrafish may be useful for studying the motor and neuronal deficits linked with amyotrophic lateral sclerosis (ALS), a study suggests. The system was described in the journal Optica, in the study “Coded-aperture broadband light field imaging using…
Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a young age, he also learned how to turn those trials into triumphs. At age 9, a stroke due to his disease left him paralyzed…
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