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Drug Therapies

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April 10, 2025
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Trofinetide improves behavioral, physical outcomes in Rett syndrome at 9 months

SAN DIEGO — Among individuals with Rett syndrome, treatment with trofinetide was linked to improvements in caregiver-reported outcomes by 9 months, according to a speaker at the American Academy of Neurology Annual Meeting.

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April 04, 2025
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Tolebrutinib slows disability accumulation in secondary progressive multiple sclerosis

SAN DIEGO — Treatment with tolebrutinib led to greater confirmed disability improvement at 6 months compared with placebo in patients with non-relapsing secondary progressive multiple sclerosis, according to research presented at AAN.

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March 07, 2025
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Fampridine may have positive impact for optic neuritis in multiple sclerosis

WEST PALM BEACH, Fla. — Treatment with fampridine may positively impact patients with multiple sclerosis who are recovering from optic neuritis, but more extensive clinical trials are required to fully assess its effectiveness, according to a poster at ACTRIMS.

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December 23, 2024
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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
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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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October 23, 2024
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EMA grants orphan drug designation to spinal and bulbar muscular atrophy drug

The European Medicines Agency has granted orphan drug designation to a small molecule intended to treat spinal and bulbar muscular atrophy, according to the manufacturer.

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October 18, 2024
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Health Canada approves Daybue for Rett syndrome in patients aged 2 years and older

Health Canada has approved Daybue to treat Rett syndrome in patients aged 2 years and older, according to manufacturer Acadia Pharmaceuticals.

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October 16, 2024
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Early initiation of Kesimpta associated with lower rates of disability worsening in MS

Early initiation and continuation of Kesimpta in those with relapsing MS was associated with lower rates of disability worsening up to 6 years compared with those who switched medication, according to a presentation at ECTRIMS 2024.

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August 02, 2024
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Lecanemab slows progression of novel Alzheimer’s-related biomarker over 18 months

PHILADELPHIA — Treatment with lecanemab slowed the progression of a potential novel Alzheimer’s disease-related biomarker by 44% over 18 months compared with placebo, with the biomarker itself most correlating with tauopathy, data show.

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June 04, 2024
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FDA clears investigational new drug application for clinical trial of ALS pill

The FDA has cleared an investigational new drug application for a phase 1/2 clinical trial to examine a novel, once-daily pill for the treatment of individuals with amyotrophic lateral sclerosis, according to the manufacturer.

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