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.

Conexiant

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."

Conexiant

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.

Conexiant

February 14, 2025

New Blood-Clotting Disorder Linked to Antibodies

Scientists have identified a chronic blood-clotting condition characterized by treatment-resistant thrombosis and linked to monoclonal antibodies targeting platelet factor 4, according to a new study.

Conexiant

February 14, 2025

Study: Patients Trust Female Docs Less

Study findings and an interview with the lead investigator.

Conexiant

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.

Conexiant

February 11, 2025

FDA Approves Mirdametinib for Neurofibromatosis Type 1

The FDA has approved mirdametinib (Gomekli), a kinase inhibitor, for adult and pediatric patients aged 2 years and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibroma...

The ASCO Post

February 10, 2025

Children With Autism and Medical Complexity Account for Disproportionate Health Care Costs

Children with both autism spectrum disorder and medical complexity have higher health care expenditures than those with autism spectrum disorder or medical complexity alone.

Conexiant

February 07, 2025

Nipah Virus Polymerase Structure Revealed

Cryo-EM reveals the structure of Nipah virus polymerase, highlighting potential antiviral targets.

Conexiant