The BMN 165 clinical development program has been designed to demonstrate the safety and efficacy of BMN 165 in reducing blood Phe concentrations in adults with PKU.
The main objectives of this large phase IIb/III paediatric study are to assess the efficacy and safety of dabigatran etexilate relative to standard of care and to document the appropriateness of the ...
This trial assessed the efficacy and safety of autologous cluster of differentiation 34 (CD34+) hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector (also called elivaldogene ...
The purpose of this trial is to determine whether treatment with valsartan will have beneficial effect in early hypertrophic cardiomyopathy (HCM) by assessing many domains that reflect myocardial ...
This proposal is to evaluate the reliability and validity of the Vanderbilt ADHD screening tool for use with adolescents and young adults aged 13-21 years. The Vanderbilt is a previously developed, ...
We plan to investigate whether the Isolated Orthosis for Thumb Actuation (IOTA) can effectively facilitate the ability of participants to perform a specific set of clinically relevant tasks conducted ...
The investigators hypothesize that Recorded Home Oximetry (RHO) utilization will not increase rates of respiratory-related re-hospitalizations and ED visits, and will not impair growth compared to ...
This study will compare three treatment regimens containing metaiodobenzylguanidine (MIBG) and compare their effects on tumor response and associated side effects, to determine if one therapy is ...
Legg-Calvé-Perthes disease is a childhood hip disorder which is common enough to be a significant public health problem (affects 1 in 740 boys between ages 0-14), but uncommon enough to have a ...
