February 21, 2025

FDA Approves First Cerebrotendinous Xanthomatosis Treatment

The FDA has approved Ctexli (chenodiol) as the first treatment for cerebrotendinous xanthomatosis, a rare lipid storage disorder.

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February 20, 2025

18-Year Neuroblastoma Remission in a Patient After GD2 CAR T-Cell Therapy

"She has never required any other therapy and is likely the longest-surviving patient with cancer who received [CAR T-cell] therapy."

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February 20, 2025

Hospital Occupancy to Reach 85% by 2035

The projection is driven by a decline in staffed beds and an aging population.

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February 19, 2025

Hospital Occupancy to Reach 85% by 2035

The projection is driven by a decline in staffed beds and an aging population.

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February 19, 2025

CRISPR Successfully Removes Extra Chromosome 21

Researchers demonstrate first successful targeted removal of extra chromosome 21 in Down syndrome cells using CRISPR-Cas9, achieving up to 30.6% effectiveness in laboratory studies.

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February 14, 2025

Study: Patients Trust Female Docs Less

Study findings and an interview with the lead investigator.

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February 12, 2025

FDA & EMA: Submitted Trial Data Discrepancies

Sample size discrepancies were identified in 13 trials (65%), with 8 differing by more than 10%.

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February 12, 2025

FDA Approves Mirdametinib for NF1-Associated Plexiform Neurofibromas

Data from the phase III ReNeu trial showed the kinase inhibitor led to an overall response rate of 41% in adult patients and 52% in pediatric patients.

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February 12, 2025

FDA Approves Evrysdi Tablet for SMA Treatment

FDA approves Roche’s Evrysdi tablet for spinal muscular atrophy, which offers a noninvasive, room-temperature stable treatment option.

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