Rare Diseases
Conference Coverage
Lentiviral gene therapy appears effective in X-CGD
HOUSTON – Results from the small study provide a “proof of concept” for the use of gene therapy in X-linked chronic granulomatous disease,...
From the Journals
Meta-analysis: IVIG bests anti-D on platelet count in pediatric ITP
But the clinical significance of this finding is unclear because of the lack of data on bleeding symptoms, according to authors of the meta-...
Daily News Podcast
Aspirin and Omega-3 fatty acids fail
Flu activity is ‘high’ in nine states.
Conference Coverage
ALL chemotherapy looks effective in mixed phenotype leukemia
SAN DIEGO – In a retrospective cohort study, most children with mixed phenotype acute leukemia achieved MRD-negative complete response on ALL-...
Daily News Podcast
Parental leave for residents
Social media can help you stay up-to-date: advice on how to use it.
News
CDC: No medical therapy can yet be recommended for acute flaccid myelitis
Updated guidelines find no solid evidence behind any treatment, all which must still be considered anecdotal.
Conference Coverage
Rituximab shines in pediatric vasculitis
CHICAGO – PePRS study provides much-needed pediatric evidence in GPA and MPA.
Conference Coverage
OV-101 shows promise for Angelman syndrome
A novel drug affecting the GABAergic system improved sleep problems in a sample of adults and adolescents with Angelman Syndrome.
News
FDA approves Sympazan for Lennox-Gastaut syndrome
Patients with Lennox-Gastaut syndrome can have difficulty swallowing pills or large-volume oral suspensions, so this oral film may reduce care...
Conference Coverage
Pediatric indications appear in one-third of orphan drug approvals
WASHINGTON – Many pediatric indications involve genetic disorders.
From the Journals
Fever, intestinal symptoms may delay diagnosis of Kawasaki disease in children