A Phase 1b clinical trial evaluating the safety and pharmacological profile of DNL343, Denali Therapeutics’ investigational oral therapy, in adults with amyotrophic lateral sclerosis (ALS) has started dosing participants. The study (NCT05006352) is currently recruiting patients at the Centre for Human Drug Research (CHDR) in Leiden, Netherlands.
Amylyx Pharmaceuticals plans to submit a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) in the coming months for approval of its investigational oral therapy AMX0035 for amyotrophic lateral sclerosis (ALS). The decision follows recent discussions with the FDA, including a pre-NDA meeting held…
A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest therapeutic needs, but one that is often left behind. Named the Institute for Life Changing Medicines, the project was…
Note: This story was updated Sept. 21, 2021, to note that 18% of patients, not 80%, experienced increases in the MUNIX(4) sum score at some point during the study. The last patient visit has been completed in Clene’s Phase 2 trial investigating the potential of CNM-Au8 — which…
A first patient has been dosed in a Phase 2 clinical trial testing Alector’s investigational antibody AL001 as a treatment for amyotrophic lateral sclerosis (ALS) associated with C9orf72 mutations. The trial is expected to enroll an estimated 45 ALS patients, who will be randomly assigned to AL001 or a placebo,…
Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running an endurance race they are highly motivated to complete, but these incremental burdens negatively impact their ability or willingness to…
A technology based on artificial intelligence is helping to spot biomarkers and document the progression of amyotrophic lateral sclerosis (ALS) in a large speech study being conducted by EverythingALS. The technology, developed by Modality.ai, is a web-based computer program that uses audio (speech) and video (facial) recordings…
ProMIS Neurosciences is advancing the development of antibodies against toxic TDP-43 protein clumps for the treatment of amyotrophic lateral sclerosis (ALS), the company announced. Through lab and animal tests, ProMIS said in a press release, the company plans to validate its current antibodies, which include extracellular antibodies delivered…
Health Canada has agreed to review Amylyx Pharmaceuticals‘ application seeking approval of AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS). “We are pleased that our submission has been accepted for review by Health Canada, and we are committed to serving those affected by ALS in the…
A $10-million grant from the National Institutes of Health (NIH) will help fund the first U.S.-based clinical trial of Synchron’s Stentrode, a wireless brain device, implanted without open brain surgery, that transmits signals from the brain directly to a computer. The COMMAND trial will evaluate whether the device…
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