The main objective of this study is to evaluate the safety and tolerability of long-term treatment with casimersen or golodirsen in patients with Duchenne muscular dystrophy (DMD).
This study evaluates the long-term safety and tolerability of tezacaftor in combination with ivacaftor (TEZ/IVA) in participants with cystic fibrosis (CF) aged 6 years and older, homozygous or ...
Study AG348-C-006 evaluated the efficacy and safety of orally administered AG-348 as compared with placebo in participants with pyruvate kinase (PK) deficiency, who were not regularly receiving blood ...
The investigators are studying the ability of a novel rapid magnetic resonance imaging (MRI) protocol to provide more accurate and earlier information about whether an infant with brachial plexus ...
1\. Design an educational quality improvement program to assess the most effective educational approach on caregiver seizure RM application. The investigators hypothesize that this educational program ...
The need for assisted-technology at home reflects some of the most serious health-related conditions faced by children with physical and developmental disabilities. 'Technology-dependent' is often ...
This proposal will evaluate an individual-use, patch-type telemetry device for simplified, single-channel EEG recording from human patients along-side the traditional in hospital wired EEG.
This study will measure plasma concentrations of dexmedetomidine, fentanyl, morphine and midazolam in pediatric patients supported with extracorporeal membrane oxygenation (ECMO) aiming to understand ...
This study will evaluate the safety and pharmacokinetics (PK) of lumacaftor (LUM) and ivacaftor (IVA) in participants 1 to less than 2 years of age with cystic fibrosis (CF), homozygous for F508del (F ...
This US study aims to assess hemophilia A patient characteristics and reasons for switching from both patient/caregiver and physician perspectives. For this purpose, this research study will include ...