Rare Diseases
Conference Coverage
Nanoparticle shows promise for ALS
A phase 2 study revealed a significant survival advantage with drug that targets mitochondria.
Conference Coverage
Brain implant is a potential life-changer for paralyzed patients
The minimally invasive device enables patients to carry out important activities of daily living.
Conference Coverage
Novel drug significantly reduces tics in Tourette syndrome – without side effects
Ecocipam does not lead to weight gain, anxiety, depression, or tardive dyskinesia, compared with placebo – a factor that may lead to better...
Conference Coverage
Gene therapy demonstrates modest success in genetic blindness
‘First step’ toward improvement in Leber hereditary optic neuropathy is maintained out to 4 years.
FDA/CDC
FDA advisory panel rejects new ALS drug
The FDA panel raised a number of issues with the clinical trial, including the study’s small sample size and no survival benefit at 24 weeks.
From the Journals
Surgery in CJD patients a potential risk factor for transmission
Literature Review
Brain implant allows fully paralyzed patient to communicate
The study shows for the first time that communication is possible in patients in a completely locked-in state and offers hope for a better quality...
Guidelines
New MIS-C guidance addresses diagnostic challenges, cardiac care
New updates in this third version of the American College of Rheumatology’s guidance include a recommendation for dual therapy with IVIg and...
Feature
Giant cell arteritis fast-track clinics pave way for greater ultrasound use in U.S.
Fast-track clinics are using vascular ultrasound to streamline diagnosis of giant cell arteritis, but the United States has a way to go to catch...
Latest News
Expected spike in acute flaccid myelitis did not occur in 2020
COVID-19 interventions may have reduced transmission of the associated enteroviruses.
Conference Coverage
Which agent is best for neuromyelitis optica?
Researchers conducted an indirect treatment comparison on the efficacy of the three FDA-approved treatment options for adults with AQP4+ NMOSD.