Rare Diseases
Conference Coverage
AI tool perfect in study of inflammatory diseases
A model that combines machine learning with clinical cues could help clinicians easily differentiate MIS-C from typhus, a new study found.
News
FDA approves first tx for rare, deadly clotting disorder
First drug to treat TTP. a rare, lethal blood clotting disorder, has been greenlighted by the FDA.
News
RNA therapeutics will ‘change everything’ in epilepsy
“We now have the potential to change the way genetic epilepsies are treated by addressing the underlying genetic cause of the disease instead of...
Conference Coverage
‘Hidden’ cognitive impairments in DMD may worsen outcomes
Long waits for neuropsychiatric evaluations make it difficult to get help, but an NIH tool may help.
Conference Coverage
Duchenne muscular dystrophy gene therapy safe, effective at 4 years
“What’s really striking, and in my mind the most impressive, is that when you follow these patients out 3 or 4 years ... you see there is this...
Latest News
Birch bark–derived treatment reduces daily dressings in patients with epidermolysis bullosa
Despite being approved in Europe, Oleogel-S10 has not yet been approved to treat EB in the United States.
News from the FDA/CDC
FDA OKs first ustekinumab biosimilar
The drug has also been granted an interchangeability designation and has been approved for six indications.
News
FDA okays drug for Duchenne muscular dystrophy
The approval of vamorolone “provides people living with Duchenne, and their families, a powerful tool to treat the disease, while limiting some...
Latest News
3-D stereophotogrammetry helps detect progression of craniofacial morphea
Existing tools that detect disease progression in patients with CM are limited.
News from the FDA/CDC
FDA approves first tocilizumab biosimilar
Tocilizumab-bavi (Tofidence) will offer a lower-cost option for patients with autoimmune conditions, the drug manufacturer, Biogen, stated.
Article
Disseminated Papules and Nodules on the Skin and Oral Mucosa in an Infant
A 38-week-old infant boy presented at birth with disseminated, nonblanching, purple to dark red papules and nodules on the skin and oral mucosa....