Rare Diseases
Conference Coverage
Hospital slashes S. aureus vancomycin resistance
LJUBLJANA, SLOVENIA – Staphylococcus aureus resistance to vancomycin is not a one-way street ending in a cliff plunge.
Latest News
NIH launches 5-year, $10 million study on acute flaccid myelitis
Researchers at the University of Alabama at Birmingham will lead the study, which aims to examine the natural history, incidence, and risk factors...
Conference Coverage
CDC: Look for early symptoms of acute flaccid myelitis, report suspected cases
The average age of a patient diagnosed with AFM is 5 years
Video
Efforts toward producing CNO/CRMO classification criteria show first results
MADRID – Cases are now being accepted for inclusion into a repository that will be used to develop and validate criteria.
Letters from Maine
A longing for belonging
Dr. Wilkoff explains how giving a patient a diagnosis fills a longing for belonging.
News
FDA approves Zolgensma for infantile-onset SMA treatment
Zolgensma is the first gene therapy for the treatment of infantile-onset spinal muscular atrophy in children aged less than 2 years.
Conference Coverage
Gentamicin restores wound healing in hereditary epidermolysis bullosa
CHICAGO - In a fatal inherited type of epidermolysis bullosa, topical gentamicin demonstrated clinical benefit.
News
Ruzurgi approved for Lambert-Eaton myasthenic syndrome in patients under age 17
The approval is the first for a LEMS treatment specifically for pediatric patients.
Conference Coverage
Angelman syndrome treatment safe, well-tolerated, and effective in exploratory analyses
PHILADELPHIA – Experimental treatment may benefit adolescents and adults with Angelman syndrome.
Conference Coverage
Neurodevelopmental concerns may emerge later in Zika-exposed infants
BALTIMORE – Some Zika-exposed children showed poorer mobility and social cognitive scores as they approached 18 months even though their fetal...
Conference Coverage
Genetic variant increases stroke risk in childhood cancer survivors
ATLANTA - Dr. Yadav Sapkota of St. Jude Children's Research Hospital discusses the identification of a genetic variant that is strongly associated...