Rare Diseases
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.
Latest News
FDA approves first once-weekly growth hormone for children
The heiGHt trial demonstrated noninferiority of lonapegsomatropin to somatropin daily injections.
FDA/CDC
FDA approves new enzyme replacement therapy for Pompe disease
The intravenous infusion supplements GAA and helps reduce glycogen accumulation.
Outcomes Research in Review
Nivolumab Plus Cabozantinib Improves Outcomes Compared With Sunitinib for Advanced Renal Cell Carcinoma
News
Two case reports identify Guillain-Barré variants after SARS-CoV-2 vaccination
Investigators in India and the United Kingdom describe rare presentations of Guillain-Barré syndrome marked by facial paralysis, but evidence of...
Conference Coverage
Two treatments show early promise for hypothalamic obesity
News
FDA panel narrowly backs avacopan approval
The FDA is mulling whether to approve avacopan for ANCA-associated vasculitis based largely on a single trial about which the agency staff has...
News
Tocilizumab (Actemra) scores FDA approval for systemic sclerosis–associated interstitial lung disease
The drug received FDA Priority Review and is the first biologic to be approved by the agency to reduce lung function decline in systemic sclerosis...
Conference Coverage
Childhood Hodgkin survivors have neurocognitive impairment