Mutations in the C9orf72 gene deregulate the process by which cells remove waste, leading to a buildup of toxic elements inside them, researchers have found. This discovery expands knowledge on how C9orf72 may contribute to the development and progression of neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS)…
Biohaven Pharmaceuticals has established an expanded access program (EAP) for patients with amyotrophic lateral sclerosis (ALS) for its investigational treatment candidate, BHV-0223, the company recently announced. The United States Food and Drug Administration regulates the expanded access program to help provide access to therapies that are currently in development and have…
My family recently made a welcome discovery. It turns out that home healthcare for ALS patients is covered by Medicare. The revelation came as we faced a dilemma: my care needs had eclipsed my wife’s capacity to provide them. We had wondered how we could be assured of…
Newly developed molecular treatment candidates called small interfering RNAs (siRNAs) have been successfully delivered to the brain and spinal cord in a rat preclinical study, Alnylam Pharmaceuticals recently announced. According to the company, the development of siRNAs may lead to the prevention or treatment of diseases such as amyotrophic…
Transplanting human spinal cord-derived neural stem cells (HSSC) into the spine of patients with amyotrophic lateral sclerosis (ALS) was found to improve their survival and function, according to clinical trial investigators. Results from the Phase 1/2 trials were compared to historical populations (controls), and the results were reported in the…
The Muscular Dystrophy Association (MDA) recently awarded the Massachusetts General Hospital (MGH) a $750,000 human clinical trial grant to study positron emission tomography (PET) imaging to measure inflammation in amyotrophic lateral sclerosis (ALS) patients as a potential biomarker. This grant, which is part of a nonprofit partnership called…
At least 800 people representing some 45 countries are soon expected to gather in Austria’s capital city, Vienna, for ECRD 2018, the 9th European Conference on Rare Diseases and Orphan Products. The May 10-12 meeting is sponsored by Eurordis, the Paris-based group that defines itself as a “patient-driven alliance”…
Patients with amyotrophic lateral sclerosis (ALS) show high prevalence of hypermetabolism, which leads to more rapid physical decline and earlier death, new study reports. The research, “Hypermetabolism in ALS is associated with greater functional decline and shorter survival,” appeared in the Journal of Neurology, Neurosurgery & Psychiatry.
Size matters when it comes to SOD1 protein aggregates found in the brains of patients with amyotrophic lateral sclerosis (ALS), according to researchers. They found that large SOD1 aggregates may help protect motor neurons from dying, while short SOD1 aggregates promote the death of motor nerve cells. Their findings support…
The ALS Association and Cytokinetics will continue to collaborate in the fight against amyotrophic lateral sclerosis (ALS) through a series of initiatives and research efforts, the organizations recently announced. As part of this partnership, biopharmaceutical company Cytokinetics is renewing its sponsorship of nationally recognized ALS Association events, including the National…
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