Rare Diseases
Conference Coverage
A farewell to arms? Drug approvals based on single-arm trials can be flawed
Objective responses, not time-dependent survival outcomes, should be endpoints for single-arm trials, with results only used for conditional...
From the Journals
Erlotinib promising for cancer prevention in familial adenomatous polyposis
After 6 months of weekly erlotinib, duodenal polyp burden was significantly reduced, with a mean percent reduction of 29.6%.
News from the FDA/CDC
FDA approves first gene therapy, betibeglogene autotemcel (Zynteglo), for beta-thalassemia
The one-time gene therapy represents a potential cure in which functional copies of the mutated gene are inserted into patients’ hematopoietic...
From the Journals
Quality of life benefit exaggerated in some cancer studies
Guidelines
Myositis guidelines aim to standardize adult and pediatric care
These are the first guidelines on how to manage adult and pediatric patients with idiopathic inflammatory myopathies (IIM).
Diminutio
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
High early recurrence rates with Merkel cell carcinoma
Approximately 95% of all recurrences happened within 3 years of the initial diagnosis in the prospective cohort study.
Rare Diseases Report 2021
Adaptive therapy borrows from nature to keep rhabdomyosarcoma in check
Researchers are applying evolutionary principles to the treatment of childhood, fusion-positive rhabdomyosarcoma.
Rare Diseases Report 2021
Mesothelioma trials: Moving toward improved survival
Latest News
Genomic screening of healthy newborns gets more popular
For now, the findings on the psychosocial effects of general newborn genomic screening show that “we should consider genetics to be just one more...
Conference Coverage
Validity of commercial serologic tests for dermatomyositis still questionable
“The testing in research laboratories is not widely available and the results are often delayed by weeks to months.”