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The United Kingdom-based Motor Neurone Disease (MND) Association was commended recently at the Third Sector Awards for its efforts in the MND community. The nonprofit organization won high commendations in the “Charity of the Year: Over £5M” category at the annual awards, which recognizes the achievements of charitable organizations and…

A French national agency has cleared AB Science to resume patient enrollment in the Phase 3 clinical trial investigating masitinib in people with amyotrophic lateral sclerosis (ALS), the company announced in a press release. AB had voluntarily suspended recruitment and treatment in all its masitinib clinical studies in…

QurAlis is advancing the development of QRL-201, its “groundbreaking” lead treatment candidate for amyotrophic lateral sclerosis (ALS), and expects to initiate its first clinical trial in late 2022. The stem cell technology company said it is now conducting formal preclinical studies to assess QRL-201’s therapeutic potential, and expects…

The two most common measures used in amyotrophic lateral sclerosis (ALS) assessments — the ALS functional rating scale-revised (ALSFRS-R) and forced vital capacity (FVC) — can predict survival more accurately when used together, according to a recent study. Alone, these measures had a similar predictive ability, but the weak…

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…