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 ...
Oxycodone is an oral opioid analgesic that is most commonly prescribed for the management of pain in post-operative patients at Boston Children's Hospital. Oxycodone has been widely used in adults and ...
The purpose of this study is to describe the range and incidence of symptoms, treatments, and complications related to pyruvate kinase deficiency (PKD). Eligible patients are those of all ages with ...
