Rare Diseases
Opinion
Dodging potholes from cancer care to hospice transitions
The consequences of suboptimal hospice transitions can damage the mental health and well-being of patient and caregiver.
Latest News
New data confirm risk of Guillain-Barré with J&J COVID shot
Nearly all individuals who developed GBS after the Janssen vaccine had facial weakness or paralysis, in addition to weakness and decreased...
Latest News
A gruesome murder changes two docs’ lives, and one was the killer
“Four years later, his patients were still shocked about what happened and couldn’t reconcile the person they knew with the event that happened...
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
Gene therapy demonstrates modest success in genetic blindness
‘First step’ toward improvement in Leber hereditary optic neuropathy is maintained out to 4 years.
News
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...
FDA/CDC
FDA approves new interferon for polycythemia vera
A longer half-life means dosing every 2 weeks, instead of weekly.
Latest News
Expected spike in acute flaccid myelitis did not occur in 2020
COVID-19 interventions may have reduced transmission of the associated enteroviruses.
News
FDA approves avacopan for rare ANCA autoimmune disease
ChemoCentryx said it plans to price its new medicine at an annual cost of $150,000-$200,000 for U.S. patients.