Rare Diseases
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.
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Lasker awardee pioneered prenatal DNA testing
With the commercial launch of noninvasive prenatal testing in 2011, health care centers around the world quickly embraced the technology as a safe...
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.
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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...
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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...
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%.