Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to help the continent’s 30 million rare disease patients and their caregivers learn about their conditions, find assistance and receive treatment. Eurordis-Rare Diseases Europe hopes to improve the current piecemeal treatment and support program with a holistic,…
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Vitamin D supplementation can help overcome low levels of the vitamin often detected in people with amyotrophic lateral sclerosis (ALS). Still, such an alternative therapeutic approach has no meaningful clinical effect on progression of ALS-associated motor symptoms, a study suggests. The study, “…
High glucose levels might compensate for the toxic effects of an accumulation of TDP-43 — a widely expressed nuclear protein that binds both DNA and RNA — and protect neurons in amyotrophic lateral sclerosis (ALS), a study in a fly model of the disease suggests. The study,…
Caregivers of people with amyotrophic lateral sclerosis (ALS) and progressive muscular atrophy (PMA) say a Dutch support program — one based on Acceptance and Commitment Therapy — empowered them to make better choices in line with their own needs, and to cope with distress, a study shows. The…
Researchers doing early-stage investigation into potential new ways to halt neuron death seen in amyotrophic lateral sclerosis (ALS) have received a grant from the Alabama Power Foundation. Rita Cowell, PhD, a researcher at Southern Research’s Drug Discovery division, and her team have identified certain compounds that in lab…
People with rare diseases know that the right government policies can make a big difference in the quality of their own lives, and those of their caregivers. But most lawmakers aren’t experts in even one well-known disease — let alone the world’s estimated 7,000 rare disorders. So how does the…
Scientist Manish Arora, PhD, received an $8 million Outstanding Investigator Award from the National Institute of Environmental Health Sciences (NIEHS) to develop models to predict and possibly prevent diseases such as amyotrophic lateral sclerosis (ALS). Arora and his team developed the Biodynamic Interface, which proposes an interplay…
Researchers at California’s Cedars-Sinai Medical Center have re-created the blood-brain barrier, a vital component of the central nervous system, using Organ-Chip technology by Emulate. This advances the possibility of patient-specific treatments for neurodegenerative disorders, including amyotrophic lateral sclerosis (ALS). Blood-brain barrier defect has been linked to…
Low levels of membralin protein in the brain cells that support and protect neurons lead to the accumulation of the neurotransmitter glutamate to levels that induce the death of motor neurons in amyotrophic lateral sclerosis (ALS), a mouse study shows. A gene therapy that rescued the levels of…
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AB Science’s New Masitinib Study Validated by EMA, With Expected Start Later This Year, Company Says
AB Science has optimized the design of a Phase 3 confirmatory study for mastinib, its oral treatment for amyotrophic lateral sclerosis (ALS), to avoid any possibility of bias. The optimized protocol for the study (NCT03127267), not yet recruiting, was validated by the European Medicines Agency (EMA). Although AB…
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