Rare Diseases
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
Shift in child hospice care is a lifeline for parents seeking a measure of comfort and hope
Under a provision of the 2010 Affordable Care Act, children enrolled in Medicaid or the Children’s Health Insurance...
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...
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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...
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...