Rare Diseases
Conference Coverage
Gene-replacement therapy shows promise in X-linked myotubular myopathy
An investigational gene therapy decreases ventilator dependence and may improve motor development in children with a rare, life-threatening...
Conference Coverage
Ataluren delays disease milestones in patients with nonsense mutation DMD
Patients who received ataluren in addition to standard of care were 2.2 years older when they lost ambulation, compared with patients receiving...
Opinion
The scope of under- and overtreatment in older adults with cancer
News
Cancer disparities: One of the most pressing public health issues
“The burden of cancer is not shouldered equally by all segments of the U.S. population,” the AACR states.
News
Drug combo slows functional decline in ALS
Patients who received the drug combination “retained higher levels of physical function over 6 months compared with those who received placebo.”...
News
COVID-19 linked to development of myasthenia gravis
Italian researchers report three cases of myasthenia gravis onset after COVID-19 infection.
FDA/CDC
FDA approves first oral treatment for spinal muscular atrophy
The oral treatment provided a “meaningful difference from the natural progression of the disease.”
News
ASCO says ‘no’ to home infusions of cancer treatment, with exceptions
The American Society of Clinical Oncology has warned against intravenous infusion of chemotherapy and monoclonal antibodies in cancer patients’...
FDA/CDC
Terlipressin squeaks by FDA review for hepatorenal syndrome 1
The FDA advisory committee’s 8-7 vote favoring approval of terlipressin for hepatorenal syndrome type 1 reflected concerns and misgivings.
News
First reported U.S. case of COVID-19 linked to Guillain-Barré syndrome
The unique clinical features in the U.S. case are urinary retention secondary to dysautonomia and ocular symptoms of...