Rare Diseases
Conference Coverage
New Drug Options Abound for Duchenne Muscular Dystrophy
Mutations are a crucial factor, neurologist says, and steroids still play a role.
From the Journals
JIA Treatment Has Increasingly Involved New DMARDs Since 2001
An analysis of a large population of commercially insured children with juvenile idiopathic arthritis in the US showed an increasing popularity of...
Conference Coverage
Pediatric Myasthenia Gravis: Don’t Treat Children Like Adults
From the Journals
Rheumatologic Disease–Associated Hyperinflammatory Condition Successfully Treated with Emapalumab
Emapalumab-containing regimens showed positive outcomes in patients with rheumatologic disease–associated hemophagocytic lymphohistiocytosis, a...
From the Journals
GI Involvement Often Present at Time of Pediatric Lupus Diagnosis or Soon After
Gastrointestinal involvement should be considered in all pediatric patients with systemic lupus erythematosus, particularly if they have abdominal...
Latest News
Neurofibromatosis: What Affects Quality of Life Most?
“To develop effective therapeutics, meaningful clinical outcomes that are tied with improvement in QoL for persons with NF1 must be clearly...
News from the FDA/CDC
Pyzchiva Receives FDA Approval as Third Ustekinumab Biosimilar
The biosimilar is approved for all indications of the reference medication, Stelara, and will launch in February 2025.
Conference Coverage
First-line Canakinumab Without Steroids Shows Effectiveness for Systemic Juvenile Idiopathic Arthritis
For JIA, a disease with limited options, a three-injection course of canakinumab was associated with durable control of disease in the absence of...
News from the FDA/CDC
FDA Approves Polyarticular JIA Indication for Sarilumab
The approval applies to patients weighing at least 63 kg, the announcement said.
News from the FDA/CDC
FDA Grants New Pediatric Arthritis Indications for Upadacitinib
The JAK inhibitor’s safety profile for patients as young as 2 years was similar to the known safety profile in adults.
News from the FDA/CDC
Belimumab Autoinjector Approved for Pediatric Lupus
This enables at-home administration of the medication, which was previously available only via intravenous infusion.