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Clinical Trials

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January 14, 2025
2 min read
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Allosteric modulator nonsuperior to placebo in Parkinson’s disease

Allosteric modulator nonsuperior to placebo in Parkinson’s disease

Treatment with an oral allosteric modulator was nonsuperior to placebo at 6 weeks in adults with Parkinson’s disease whose symptoms included fall risk and cognitive issues, data from JAMA Neurology show.

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January 13, 2025
1 min read
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Early blarcamesine use slows AD progression over 3 years

Early blarcamesine use slows AD progression over 3 years

Topline results from a phase 2b/3 clinical trial of blarcamesine for individuals with early-stage Alzheimer’s disease showed that earlier initiation of the oral therapeutic led to significant disease slowing over 3 years.

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January 08, 2025
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Novel small molecule for ALS fails to meet primary endpoint in phase 2/3 study

Novel small molecule for ALS fails to meet primary endpoint in phase 2/3 study

Topline results from a phase 2/3 clinical trial showed that an eIF2B agonist to treat ALS failed to meet its primary endpoint of slowing disease progression compared with placebo at week 24.

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January 03, 2025
1 min read
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Gene therapy slows progression of spinal muscular atrophy type 2

Gene therapy slows progression of spinal muscular atrophy type 2

Topline results from a phase 3 clinical trial showed an investigational gene therapy for young people with spinal muscular atrophy type 2 increased mobility and slowed disease progression, according to a press release from Novartis.

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December 30, 2024
21 min listen
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The Role of Machine Learning in Clinical Trials with Damon Rasheed

The Role of Machine Learning in Clinical Trials with Damon Rasheed

In this episode, host Shikha Jain, MD, speaks with Opyl co-founder Damon Rasheed about using machine learning to better predict clinical trial outcomes, handling ethical concerns surrounding AI in health care and more.

News
December 23, 2024
1 min read
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Oral small molecule shows promise in dementia with Lewy bodies

Oral small molecule shows promise in dementia with Lewy bodies

According to results from the exploratory phase 2 SHIMMER clinical trial, a small molecule oligomer antagonist improved outcomes in patients with dementia with Lewy bodies.

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December 23, 2024
2 min read
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NG101 nonsuperior to placebo at 6 months in cervical spinal cord injuries

NG101 nonsuperior to placebo at 6 months in cervical spinal cord injuries

For individuals with recent acute cervical spinal cord injuries, treatment with a novel neuroregenerative therapeutic was nonsuperior to placebo after 6 months, according to research published in The Lancet Neurology.

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December 20, 2024
4 min watch
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VIDEO: Zorevunersen significantly reduces seizures in Dravet syndrome

VIDEO: Zorevunersen significantly reduces seizures in Dravet syndrome

LOS ANGELES — Zorevunersen led to 75% to 80% seizure reduction in patients with Dravet syndrome, according to Barry S. Ticho, MD, PhD, FACC, chief medical officer of Stoke Therapeutics.

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December 05, 2024
2 min watch
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VIDEO: Bringing patient voices to the forefront in rheumatoid arthritis clinical trials

VIDEO: Bringing patient voices to the forefront in rheumatoid arthritis clinical trials

WASHINGTON — In this video, Micaela F. Bayard, MD, discusses the shift at major conferences to include patients voices and perspectives.

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December 03, 2024
1 min read
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Simufilam fails to meet endpoints in phase 3 Alzheimer’s study, trial discontinued

Simufilam fails to meet endpoints in phase 3 Alzheimer’s study, trial discontinued

Topline results from the phase 3 ReThink-ALZ study of simufilam in mild to moderate Alzheimer’s disease showed the investigational therapeutic did not meet any of its prespecified endpoints.

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