News

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…

New technology that tests therapies designed for disorders of the neuromuscular junction, such as amyotrophic lateral sclerosis (ALS), closely mimics the human condition and offers a new method of drug testing, according to a report. The paper, “Stem cell derived phenotypic human neuromuscular junction model for…

MediciNova’s investigational therapy MN-166 (ibudilast) was able to delay disease worsening and improve survival in amyotrophic lateral sclerosis (ALS) patients, updated data from a Phase 2 clinical trial show. The study, “Ibudilast — Phosphodiesterase Type 4 Inhibitor — Bi-Modal Therapy with Riluzole in Early [Not…

Investigational therapy NP001 failed to improve disease severity and pulmonary function in a Phase 2 confirmatory trial for amyotrophic lateral sclerosis (ALS) patients with elevated levels of systemic inflammation, Neuraltus Pharmaceuticals announced. The findings, showing the trial failed to meet its primary and secondary goals, were recently shared during the…