Rare Diseases
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
FDA/CDC
FDA clears first drug for rare genetic causes of severe obesity
Setmelanotide (Imcivree) is indicated for weight management in adults and children as young as 6 years with severe obesity driven by genetic...
Article
Using telehealth to deliver palliative care to cancer patients
Dr. Alan Lyss discusses the use of telehealth for palliative cancer care, including results from the REACH PC trial.