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Rare Diseases

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November 25, 2025

FDA OKs First SMA Gene Replacement Therapy

The FDA approved Itvisma, the first gene replacement therapy for children 2 years and older, teens, and adults with spinal muscular atrophy, a one-time treatment that replaces the SMN1 gene and demonstrated improved motor function in Phase 3 studies.

Conexiant

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November 25, 2025

Brain Shifts at Hidden Ages 

Manifold analysis reveals distinct organizational epochs separated by turning points at ages 9, 32, 66, and 83 years across 4,216 diffusion imaging scans.

Conexiant

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November 19, 2025

What the Storms Left Behind Was Worse

Severe storm-related mold infestations are contributing to ongoing respiratory and systemic health risks amid significant delays in remediation and disaster recovery support.

KFF Health News

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November 18, 2025

Plozasiran Wins FDA Nod for FCS

Arrowhead’s small interfering RNA therapy reduced triglycerides and lowered pancreatitis risk in the phase 3 PALISADE trial, providing a quarterly self-administered option for patients with familial chylomicronemia syndrome.

Conexiant

Daily News

Stay up to date with the latest clinical headlines and other information tailored to your specialty.

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November 13, 2025

‘FDA Said Yes, Despite Inconclusive Evidence’

The FDA’s authorization of the Q-Collar is under scrutiny after researchers identified duplicated data, altered study endpoints, and unsubstantiated “brain protection” claims.

Conexiant

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November 11, 2025

Advancing Gene Therapy for Canavan Disease

Interim results from a clinical trial addressing a fatal brain matter disorder suggest that gene therapy could be an effective approach

The Medicine Maker