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May 29, 2020May 29, 2020

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

News, syndicated

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene ... Read more

May 29, 2020May 29, 2020

RESCUE-ALS Trial Enrolls More Than Half of Patients Ahead of Schedule

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The Phase 2 clinical trial investigating Clene Nanomedicine‘s CNM-Au8 as a potentially disease-modifying therapy for people with amyotrophic lateral sclerosis (ALS) has enrolled more than half of its participants ahead of schedule. RESCUE-ALS ... Read more

May 27, 2020May 27, 2020

Mitsubishi Tanabe Opens Phone Support for Those Interested in Radicava

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Mitsubishi Tanabe Pharma America (MTPA) has set up a support program for adults living with amyotrophic lateral sclerosis (ALS) and wanting to learn more about this disease and Radicava (edaravone), ... Read more

May 26, 2020May 29, 2020

Arimoclomol Earns FDA’s Fast Track Status for ALS

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The U.S. Food and Drug Administration (FDA) has given fast track designation to Orphazyme’s investigational therapy arimoclomol for the treatment of amyotrophic lateral sclerosis (ALS). A therapy candidate is put on the ... Read more

May 25, 2020May 25, 2020

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

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European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session ... Read more

May 22, 2020May 22, 2020

High Creatinine Kinase Levels Predict Slower Progression in ALS, Study Finds

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Levels of creatinine kinase — a marker of muscle damage — are elevated in most amyotrophic lateral sclerosis (ALS) patients and appear to be significantly higher among those whose disease progresses ... Read more

May 21, 2020May 21, 2020

Gut Microbiota Affects Inflammation and Lifespan in ALS, Mouse Study Shows

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Changing the composition of bacteria in the gut using antibiotics or fecal transplants may help prevent or reduce the severity of symptoms of amyotrophic lateral sclerosis (ALS), a study ... Read more

May 20, 2020

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

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Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. ... Read more

May 20, 2020May 20, 2020

Cerebrospinal Fluid May Transport ALS Toxins, Mouse Study Suggests

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The clear, colorless liquid that fills and surrounds the brain and spinal cord may spread toxic protein aggregates from one nerve cell to others among people with amyotrophic lateral ... Read more

May 19, 2020May 19, 2020

Neurotoxin of Blue-Green Algal Blooms May Raise Risk of ALS

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Exposure to a neurotoxic molecule produced by blue-green algae seems to raise a person’s odds of developing amyotrophic lateral sclerosis (ALS), particularly for those under 65, a population-based study ... Read more

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