Rare Diseases
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...
Latest News
Topical gene therapy for dystrophic epidermolysis bullosa shows promise
B-VEC was well tolerated with no drug-related serious adverse events or discontinuations due to treatment.
Latest News
Topical gel for epidermolysis bullosa shows ongoing benefit
Earlier this year, the FDA declined to approve oleogel-S10 for use in EB, even after it extended its review by 3 months to include additional...
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.
Latest News
Pandemic public health measures may have mitigated Kawasaki disease
Cohort study data suggest that actions to reduce the spread of COVID-19 also reduced exposure to Kawasaki disease triggers.
News
FDA approves setmelanotide for obesity in Bardet-Biedl syndrome
The FDA cleared setmelanotide (Imcivree) for people age 6 and older with obesity due to a rare genetic disorder.