Rare Diseases
Conference Coverage
Cerliponase alfa continues to impress for CLN2 disease
BANGKOK – The therapy durably slowed decline in motor and language function seen after 3 years of treatment.
Conference Coverage
‘Pot’ is still hot for Dravet, Lennox-Gastaut
BANGKOK – Long-term extension studies show sustained, consistent seizure reductions.
From the Journals
Possible role of enterovirus infection in acute flaccid myelitis cases detected
High levels of enterovirus peptides were found in the cerebral spinal fluid of individuals with AFM.
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.