Rare Diseases
Conference Coverage
C-Path and NORD team up to speed development of treatments for rare disorders
ROCKVILLE - A new data analytics platform aims to accelerate therapies from bench to bedside for 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.
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...
From the Journals
Study refines ALS risk among first-degree relatives of patients with disease
Heritability was high, even in the absence of known genetic 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
Latest News
FDA approves first treatment for neuromyelitis optica spectrum disorder
The Food and Drug Administration has approved Soliris (eculizumab) injection for IV use for the treatment of neuromyelitis optica spectrum...
From the Journals
Novel genetic therapy reduces key protein in Huntington’s disease
Intrathecal therapy was associated with dose-dependent reductions in mutant huntingtin protein, but clinical change will require further study.
Conference Coverage
Mutant huntingtin and neurofilament light are potential biomarkers in Huntington’s disease
PHILADELPHIA – The biomarkers successfully distinguish between healthy controls, patients with premanifest disease,...
FDA/CDC
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.