Clinical Trials

Alzheon Inc. has announced positive interim findings from an ongoing phase 2 study evaluating ALZ-801, an investigational disease-modifying treatment for Alzheimer’s disease.

Delandistrogene moxeparvovec improved motor function and was well-tolerated 4 years after treatment in a small cohort of children with Duchenne muscular dystrophy, according to a poster at the 2023 MDA Clinical & Scientific Conference.

Ataluren preserved muscle burst activity across a range of ambulatory tests compared with placebo in boys with nonsense mutation Duchenne muscular dystrophy, according to a poster at the 2023 MDA Clinical & Scientific Conference.

A clinical biopharmaceutical company has announced completion of the clinical portion of its phase 2a multiple ascending dose study examining safety and tolerability of AL001 for dementia related to Alzheimer’s disease.

Treatment with casimersen increased exon 45 skipping from baseline and was well-tolerated in children with Duchenne muscular dystrophy, according to a poster at the 2023 MDA Clinical & Scientific Conference.

A biopharmaceutical company has announced positive data from its phase 3 open-label study evaluating the safety and efficacy of intranasal pherine fasedienol for adults diagnosed with social anxiety disorder.

Ravulizumab was associated with fewer clinical deterioration events in adults with anti-acetylcholine receptor antibody-positive generalized myasthenia gravis, according to a poster at the 2023 MDA Clinical & Scientific Conference.

Treatment with givinostat was associated with improvement in mobility and physical function compared with placebo in ambulant boys with Duchenne muscular dystrophy, according to a poster at the 2023 MDA Clinical & Scientific Conference.

A biopharmaceutical company has announced positive topline results from its phase 2, randomized, open-label trial of evenamide as an add-on to antipsychotic medication in those with moderate to severe treatment-resistant schizophrenia.

Stoke Therapeutics Inc. announced the FDA will allow administration of a higher single dose of its investigational drug STK-001 in its ongoing phase 1/2a MONARCH study of children and adolescents with Dravet syndrome.