Rare Diseases
News
More data back Guillain-Barré risk with Janssen COVID shot
Observed GBS cases after the Janssen shot were 2 to 3 times greater than expected, based on background rates within 21 and 42 days of vaccination...
News
Cancer clinics begin to accommodate patients demanding new cancer detection tests
Sales teams are making a big push for multicancer early detection tests.
News
Have you heard of VEXAS syndrome?
The prevalence of this syndrome is unknown, but it is not so rare.
News
High drug costs exclude most neurology patients from cutting-edge treatment
“Our study of people with neurologic conditions found that fewer than 20% were being treated with new medications.”
Conference Coverage
Diazepam nasal spray effective in Lennox-Gastaut syndrome
A post hoc analysis shows that the drug works about as well in Lennox-Gastaut syndrome as in patients with pediatric encephalopathies.
FDA/CDC
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.
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...
News
Quality of life benefit exaggerated in some cancer studies
Latest News
Acute hepatitis cases in children show declining trend; adenovirus, COVID-19 remain key leads
Adenovirus remains the most commonly detected virus in acute hepatitis in children, found in 53% of cases overall.
FDA/CDC
FDA approves oral form of ALS drug edaravone
Compared with the IV formation of the drug, the oral form has been shown to generate comparable levels of active drug in the bloodstream.