Rare Diseases
Latest News
AAP guidance helps distinguish bleeding disorders from abuse
Laboratory testing of bleeding cannot always rule out abuse, just as a history of trauma may not rule out a bleeding disorder or other medical...
From the Journals
Ketamine promising for rare condition linked to autism
Using parent-report instruments to assess treatment effects, ketamine was associated with “nominally significant” improvement in a variety of...
News from the FDA/CDC
CDC warns of enterovirus strain linked to polio-like condition
The advisory also urged providers to “strongly consider AFM in patients with acute flaccid limb weakness, especially after respiratory illness or...
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...
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
Biosimilar-to-biosimilar switches deemed safe and effective, systematic review reveals
While switches between biosimilars of the same reference biologic already occur for biosimilars with indications in different specialties, they do...
Latest News
Two deaths from liver failure linked to spinal muscular atrophy drug
These are the first known fatal cases of acute liver failure associated with the drug, which the company notes was a known side effect included in...
From the Journals
Stopping JIA drugs? Many can regain control after a flare
About 67% of patients who stopped medications for well-controlled JIA got their condition under control by 1 year after postflare treatment,...
News from the FDA/CDC
Ustekinumab becomes second biologic approved for PsA in kids
The approval is based on an extrapolation of data from adult trials in active psoriatic arthritis and adult and pediatric studies in moderate to...
News from the FDA/CDC
FDA approves belimumab for children with lupus nephritis
Children with lupus-related kidney damage can now receive the B cell–reducing therapy that adults with lupus nephritis have been able to get for...