SIRION Biotech and Denali Therapeutics have signed a new licensing agreement to develop a new generation of adeno-associated virus (AAV) vectors, harmless viruses used as vehicles to deliver gene therapies, to allow therapies to reach into the central nervous system (CNS) of people with amyotrophic lateral sclerosis (ALS). The…
An upcoming webinar will focus on the novel approach to clinical trial design in the Modifying Immune Response and Outcomes in ALS (MIROCALS) trial, which aims to break the current impasse in therapy development for amyotrophic lateral sclerosis (ALS). During the webinar, P. Nigel Leigh, a motor neuron disease…
The majority of patients with amyotrophic lateral sclerosis (ALS) are excluded from participating in clinical trials, a large study has revealed. This finding raises questions on the extrapolation of trial results for this patient population. The researchers also stressed the need for individualized risk-based criterion to balance the gains in…
Harvard neuroscientists have discovered how the TDP-43 protein — previously linked to sporadic and inherited amyotrophic lateral sclerosis (ALS) cases — reduces the levels of a second player, called stathmin2 (STMN2) — which is necessary for neuron growth and regeneration — making the STMN2 gene a potential new therapeutic target. The study, “…
Treatment with ultra-high-dose methylcobalamin, the physiologically active form of vitamin B12, may improve the prognosis of patients with amyotrophic lateral sclerosis (ALS) who receive it a year or less after symptom onset, a…
A $300,000 grant from the Muscular Dystrophy Association (MDA) will back AcuraStem’s preclinical development of an amyotrophic lateral sclerosis (ALS) patient-derived stem cell therapy that has the potential to treat a wide range of ALS patients. The grant will allow the biotechnology company AcuraStem to begin proof-of-concept studies of its…
A human-derived antibody that specifically binds to abnormal superoxide dismutase 1 (SOD1) improved the symptoms and delayed disease progression in a mouse model for amyotrophic lateral sclerosis (ALS), a study shows. The antibody, alpha-miSOD1, could be further developed as a candidate treatment for ALS caused by SOD1 misfolding. The study titled…
EnClear Therapies, a biotech company developing a device to halt the progression of neurological diseases including amyotrophic lateral sclerosis (ALS), announced it has spun out from QurAlis, one of its founding companies.
The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Revalesio’s experimental therapy RNS60 for people diagnosed with amyotrophic lateral sclerosis (ALS). A phase 2 trial is now recruiting participants. Fast Track designation is intended to shorten the time it takes for the…
Difficulty in recognizing emotions may be a sign of changes in the frontal lobe of the brain and associated behavioral symptoms in patients with amyotrophic lateral sclerosis (ALS). The study with that finding, “Deficits in Emotion Recognition as Markers of Frontal Behavioral Dysfunction in Amyotrophic Lateral Sclerosis,”…
