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Rhizomelic chondrodysplasia punctata (RCDP) is a group of rare diseases that have no known cure. It is a genetic disorder characterized by mutations in the Peroxisomal Biogenesis Factor 7 (PEX7) gene (RCDP1), Glyceronephosphate O-acyltransferase (GNPAT) gene (RCDP 2), Alkylglycerone phosphate
Study design This was a randomised, double-blind, placebo-controlled clinical trial among children with a presumptive diagnosis of acute migraine in the ED. The study was approved by the institutional IRB and written informed assent and consent were obtained by the patient and a parent respectively,
The success of BMT as a curative option for patients with malignancies is frequently limited by the inability to identify an appropriate donor in time for transplantation.
Transplantations utilizing umbilical cord blood stem cells are increasingly successful. Data suggest that there are fewer and
This is a phase 2 trial to evaluate the safety and effectiveness of a new immunosuppressive drug, sirolimus, in patients with idiopathic and lupus membranous nephropathy. Patients (age greater than or equal to 13 years) will be invited to participate if they have persistent nephrotic range