Philadelphia May 20, 2025 – A collaborative team of researchers from Children’s Hospital of Philadelphia (CHOP) found that hematopoietic stem cell transplantation (HSCT) is a highly effective method ...
Misdiagnosis or underdiagnosis of rare diseases in patients with diagnoses of common diseases can lead to delayed or inappropriate treatments, thereby complicating the management of both rare and ...
Hypertension is an important public health problem affecting more than 50 million individuals in the US alone. The most common form, essential hypertension, results from the complex interplay between ...
The increased kidney failure risk among patients with monogenic kidney disorders supports genetic testing in routine clinical practice and research on potential new therapies. Patients with monogenic ...
Genetic research uncovers a continuum between diabetes forms, offering insights for precision medicine. Study: Rare variant analyses in 51,256 type 2 diabetes cases and 370,487 controls reveal the ...
Immunosuppressants used in adeno-associated virus (AAV) gene therapy for monogenic disorders present an adverse effect profile that is consistent with high-dose steroid use in other conditions, ...
Allogeneic HSCT achieved 92% sustained, medication-free remission and 100% survival in children with monogenic IBD, highlighting its potential as a transformative treatment. Monogenic IBD, ...
An international team of researchers has made a key discovery: many children and young adults in Sub-Saharan Africa diagnosed with type 1 diabetes (T1D) may have a different form of the disease—one ...
Replacing rogue microglia with healthy ones can ameliorate, in a mouse model, a childhood neurodegenerative disease called Globoid cell leukodystrophy (GLD), aka Krabbe disease. The approach even ...
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