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April 12, 2023
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FDA grants fast track designation for novel Duchenne muscular dystrophy gene therapy

FDA grants fast track designation for novel Duchenne muscular dystrophy gene therapy

Regenxbio Inc. announced that the FDA has granted fast track designation for RGX-202, a one-time gene therapy for treatment of Duchenne muscular dystrophy.

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April 11, 2023
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Phase 1/2a trial commenced for Alzheimer’s dementia immunotherapy

Phase 1/2a trial commenced for Alzheimer’s dementia immunotherapy

Alzamend Neuro Inc. announced initiation of a phase 1/2a clinical trial for its proprietary immunotherapy vaccineALZN002 to treat mild to moderate dementia associated with Alzheimer’s disease.

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April 10, 2023
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First patient dosed in phase 1/2a trial of Huntington’s, spinocerebellar ataxia therapy

First patient dosed in phase 1/2a trial of Huntington’s, spinocerebellar ataxia therapy

Vico Therapeutics announced that the first patient has been dosed in a phase 1/2a study evaluating VO659, an investigational antisense oligonucleotide for Huntington’s disease and spinocerebellar ataxia types 1 and 3.

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April 10, 2023
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VIDEO: Diversity in clinical trials for psoriasis emphasized at AAD 2023

VIDEO: Diversity in clinical trials for psoriasis emphasized at AAD 2023

In this Healio exclusive video, Laura Korb Ferris, MD, discusses the importance of assessing racial and ethnic diversity in phase 3 clinical trials of risankizumab for the treatment of plaque psoriasis.

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April 06, 2023
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Phase 1b study will evaluate small molecule adenosine receptor agonist for stroke, TBI

Phase 1b study will evaluate small molecule adenosine receptor agonist for stroke, TBI

Astrocyte Pharmaceuticals Inc. announced initiation of a phase 1b study of AST-004, a small molecule adenosine A3/A1 receptor agonist for stroke and traumatic brain injuries that will be evaluated in healthy volunteers.

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April 03, 2023
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VIDEO: Research on geographic atrophy should focus 'everywhere'

VIDEO: Research on geographic atrophy should focus 'everywhere'

Healio spoke with Yasha Modi, MD, to discuss research needs in geographic atrophy.

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April 03, 2023
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Positive phase 2 results reported for novel combination therapy for Parkinson’s disease

Positive phase 2 results reported for novel combination therapy for Parkinson’s disease

CuraSen Therapeutics Inc. announced positive data from its randomized, double-blind crossover phase 2 study of CST-103/CST-107, which demonstrated improvements in cognition and mood in those with Parkinson’s disease.

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March 31, 2023
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Phase 3 study to assess safety, efficacy of Huntington’s disease therapeutic complete

Phase 3 study to assess safety, efficacy of Huntington’s disease therapeutic complete

Prilenia Therapeutics announced the completion of its global phase 3 study of pridopidine for Huntington’s disease, with topline results expected in the next few months.

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March 30, 2023
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Topline results announced from phase 2 trial of treatment for early Parkinson’s disease

Topline results announced from phase 2 trial of treatment for early Parkinson’s disease

Neuraly has announced that its investigational NLY01 did not meet the primary study endpoint of improvement in motor symptoms in patients with early, untreated Parkinson’s disease.

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March 30, 2023
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Pitolisant safe, improved symptoms of narcolepsy in children

Pitolisant safe, improved symptoms of narcolepsy in children

Treatment with pitolisant demonstrated significant efficacy in reducing excessive daytime sleepiness and cataplexy in children aged 6 years and older, according to research presented in The Lancet Neurology.

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