Rare Diseases
Latest News
Combination of energy-based treatments found to improve Becker’s nevi
The study is the largest known case series of therapy combining 1,550-nm nonablative fractional resurfacing for patients with Becker’s nevus with...
From the Journals
IVIG proves effective for dermatomyositis in phase 3 trial
Prior to this trial, intravenous immune globulin had not been fully studied for the treatment of dermatomyositis.
Opinion
Cancer as a full contact sport
The complexity of cancer care has long necessitated a team approach to planning cancer treatment.
News
ALS drug gets FDA panel thumbs-up after rare second look
FDA officials in the meeting stressed the importance of considering unmet medical need in ALS in the panel’s decision-making process.
Feature
Increasing primary care doctors’ knowledge of IPF could speed up diagnoses, suggests white paper
Nonspecific symptoms of IPF may prompt primary care providers to pursue other assessment pathways.
Latest News
Uncombable hair syndrome: One gene, variants responsible for many cases
A deficiency in the shaping and mechanical strengthening of the hair shaft occurs in the UHS phenotype, which is characterized by dry, frizzy, and...
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...
Feature
ILD on the rise: Doctors offer tips for diagnosing deadly disease
The COVID-19 pandemic has added a new challenge to making the diagnosis of interstitial lung disease, Dr. Kulkarni said. COVID can lead to...
News from the FDA/CDC
FDA okays spesolimab, first treatment for generalized pustular psoriasis
The drug received priority review and had orphan drug and breakthrough therapy designation.
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...