The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. The event’s sponsor, the National Organization for Rare Disorders (NORD),…
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A single brain area previously thought to control only the movement of the hands and arms also may control movement across all four limbs, a study found. This discovery opens new avenues for the use of brain-computer interfaces in people who have lost control of their arms and legs,…
People with amyotrophic lateral sclerosis (ALS) are less likely to have used anti-diabetic medications in the five years preceding their diagnosis than matched individuals of the same population, a study found, supporting an inverse association between diabetes treatments and the risk of ALS. The research also points to…
Radicava (edaravone), an approved treatment for amyotrophic lateral sclerosis (ALS), can be taken for up to one year without losing its effectiveness, and benefits patients who delay its start — say, after six months of placebo in a clinical trial, a post hoc analysis of that Phase 3…
Tikomed has secured $5.5 million in financing from investors to support the ongoing clinical development of ILB, its lead therapy candidate for degenerative neurological diseases, including amyotrophic lateral sclerosis (ALS). The funding also will help develop Ibsolvmir, a therapy to increase the effectiveness of transplanting cells to treat patients…
Target ALS and The ALS Association will jointly fund two projects focused on the development of new tests to evaluate TDP-43’s potential as a biomarker of amyotrophic lateral sclerosis (ALS). Nearly all ALS cases are characterized by the formation of toxic aggregates (clumps) containing the TDP-43 protein. These…
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‘Hero’ Proteins Protect Cells from Toxic Clumps Underlying ALS, Other Neurodegenerative Diseases
Human cells produce a type of proteins, termed “Heroes,” that protect against the formation of toxic protein aggregates involved in several neurological conditions, including amyotrophic lateral sclerosis (ALS), and are extremely resistant to harsh conditions, early study results show. Organisms naturally use Hero proteins as molecular shields to stabilize…
Levels of specific proteins found in specialized immune cells — white blood cells like lymphocytes and monocytes — can serve as biomarkers for amyotrophic lateral sclerosis (ALS), helping doctors to diagnose the disease and its likely progression, a study reported. These specialized immune cells, called peripheral blood…
Bloom Science, a biopharmaceutical company, announced it has received a $500,000 grant from The ALS Association to advance a treatment that, by managing bacteria populations in the gut, might restore neurotransmitters in the brain to ease motor symptoms of…
Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see the approval of 1,000 new rare disease therapies by 2027. That group, the International Rare Diseases Research Consortium (IRDiRC) —…
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