FDA approved levacetylleucine (Aqneursa) for ataxia in ataxia-telangiectasia, the first therapy for the disease, in patients 15 kg or heavier.
|Articles|January 1, 2005
Severe brain injury: Helping patient and family on the long road back
Traumatic brain injury affects virtually every aspect of the patient's-and family's-life. Here's what to expect in the aftermath and how to help the family deal with the often overwhelming demands of rehabilitation and return to home and community.
Advertisement
Advertisement
Related to this article
FDA approved apitegromab-mstn (Isembyld) for SMA in patients 2 years and up, based on phase 3 data showing added motor gains with SMN2 therapy.
FDA granted priority review to Teva's ecopipam for pediatric Tourette syndrome, backed by phase 3 data showing reduced relapse risk.
Tallie Z. Baram, MD, talks about how adding a measure of childhood unpredictability to ACEs screening may help pediatricians identify children at risk for mental health problems.
Review some of the top stories from the Contemporary Pediatrics website over the last week, and catch up on anything you may have missed.
The FDA granted priority review to efzimfotase alfa for HPP in patients aged 2 years and older; a decision is expected in the first half of 2027.
Adolescent VALIANT subgroup data show pegcetacoplan cut proteinuria 75% vs placebo, supporting its expanded pediatric kidney indication.
The FDA approved Fayuvi (rebisufligene etisparvovec-hopf), a one-time AAV9 gene therapy, as the first treatment for pediatric MPS IIIA (Sanfilippo syndrome type A).
Advertisement
Advertisement
Trending on Contemporary Pediatrics
1
Updated AAP guidance changes iron deficiency screening and treatment in children
2
How Breast Milk Shapes Gut Microbiome Signaling and Immunity
3
Tezepelumab meets co-primary and secondary endpoints in phase 3 eosinophilic esophagitis trial
4
Pegcetacoplan reduces proteinuria, stabilizes kidney function in adolescents with C3G and IC-MPGN
5