This study wants to learn if breathing tests (called spirometry) performed in an outpatient clinic give results that are as reliable and accurate as spirometry that is done in a specialized lung function lab.
This study wants to learn more about how blood sugar levels in healthy children without chronic disease compare to the blood sugar levels of children with Cystic Fibrosis (CF). We are only recruiting children without chronic disease to join the study.
This study wants to look at how endocannabinoids are different in children with and without autism. We are only looking for children who do not have autism to join our study.
An Open-Label multinational, multicenter study to evaluate the long-term safety, tolerability, and efficacy of subcutaneous amlitelimab in participants aged 12 years and older with moderate to severe atopic dermatitis
A Phase 2, Randomized, Human Growth Hormone-Controlled, Multicenter, Basket Study of Vosoritide in Children with Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome with an Inadequate Response to Human Growth Hormone
To evaluate the safety and tolerability of benralizumab
This study wants to collect health and growth information about children with hypochondroplasia. This information will help us understand their basic health and see if they can join future studies about hypochondroplasia.
The primary objectives of DISCOVERY are to: 1. Identify the physiologic drivers and other determinants of progression to youth-onset T2D by charting the course of changes in glycemia, β-cell function, insulin sensitivity, psychosocial, behavioral, and social determinants of health during this critical period of maturation 2. Inform approaches for precise prediction of which youth are at highes...
To determine the recommended phase 2 dose (RP2D) of SNDX-5613 administered in combination with chemotherapy in patients with relapsed or refractory (R/R) KMT2A-rearranged (KMT2A-R) acute lymphoblastic leukemia (ALL).
This study will evaluate the effectiveness and safety of risdiplam (Evrysdi®) administered to patients under 2 years of age genetically diagnosed with spinal muscular atrophy (SMA) and 2 SMN2 copies who have previously received onasemnogene abeparvovec and experience a plateau or decline in function.
study on the efficacy, safety and pharmacokinetics of Debio 4326 in pediatric participants who are receiving gonadotropin-releasing hormone agonist therapy for central precocious puberty
Treatment of Duchenne Muscular Dystrophy (DMD) with dystrophin deletion amenable to exon 50 skipping
To assess the safety and tolerability of ferric citrate in pediatric subjects with hyperphosphatemia related to chronic kidney disease (CKD).
To assess the safety and tolerability of EDG-5506 in children with DMD
To determine the effect of abnormal menstruation on markers of cardiovascular disease in adolescent athletes.
We are studying teens who feel angry a little, sometimes, or a lot to see if they think about social situations in different ways. Teens who often feel very angry might have more problems when they grow up, so we want to learn more about how their brains work.
Why should I join this research study? We want to help prepare for future pandemics by learning more about infections that can cause people to become sick, like Enterovirus D68, and how a person may respond and fight off a potential infection
The objective of this study is to follow, characterize and evaluate the natural history of the disease course, burden of illness, risk factors, quality of life (QoL), and biomarkers associated with disease progression and treatments in pediatric patients with cardiomyopathy (CM) due to MYBPC3 pathogenic or likely pathogenic mutations.
Aim 1: Examine the efficacy of a group cognitive behavioral treatment (CBT), Facing Your Fears: Autism Spectrum disorder/Intellectual Disability (FYF: ASD/ID) on anxiety in a randomized controlled trial (RCT). Participants will be randomized to FYF: ASD/ID or a treatment as usual (TAU) group. Aim 2: Examine secondary outcomes such as how emotion regulation, problem behavior, parenting self...
To evaluate the safety and tolerability of multiple-ascending dose (MAD) levels, 40 mg/kg, 60 mg/kg, and 80 mg/kg, of NS-089/NCNP-02 in ambulant boys with DMD after 4 weeks of once weekly intravenous (IV) treatment; and o evaluate the pharmacokinetics (PK) of MAD levels of NS-089/NCNP-02.
To understand the effects of puberty blockers and puberty on insulin sensitivity, metabolism and vascular health
We are purposing this retrospective and prospective, multicenter observational study in order to facilitate quality improvement and description of treatment with this novel dialysis device dedicated to the treatment of infants with AKI or fluid overload.