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This is a 2-part, prospective, open-label, single arm, multicenter study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PDx), lenolisib在至少15例(1 - 6岁)患有活化磷酸肌肽3-激酶δ (PI3Kδ)综合征(APDS)的儿童患者中的疗效

本研究的主要目的是评估在12个月的治疗期内，每天给予250微克(mcg) DBV712诱导4-7岁花生过敏儿童对花生脱敏的有效性和安全性.

本研究的总体目标是为先天性1型肌强直性营养不良儿童和儿童期1型肌强直性营养不良儿童建立有效的临床终点评估, and develop biomarkers for the condition.

This is an international, multi-center, randomized, prospective, double-blind, placebo-controlled, Phase 3 study, 目的:评价EryDex(地塞米松磷酸钠[DSP])对自体红细胞的影响。, administered by intravenous (IV) infusion once every 28 days, on neurological symptoms of patients with Ataxia Telangectasia (A-T).

This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single IV infusion of SGT-003 in participants with Duchenne muscular dystrophy. There will be 2 cohorts in this study, dosed sequentially. Cohort 1 will include participants 4 to <6 years of age, inclusive. Cohort 2 will only be opened after dosing and monitoring a subset of participants in Cohort 1. Cohort 2 will include participants 6 to <8 years of age, inclusive. 所有参与者将接受SGT-003，并将参加为期5年的长期随访研究.

本临床研究的目的是进一步了解奥贝德西韦(obeldesivir, ODV)治疗2019冠状病毒病(COVID-19)儿童和青少年的安全性和耐受性。.

The primary objectives are to evaluate the plasma pharmacokinetics (PK), safety and tolerability of ODV in pediatric participants with COVID-19.

本研究的目的是提供一项单一的临床研究，以统一的方法监测在先前的临床研究中接受delandistrogene moxeparvovec的参与者的长期安全性和有效性. No study drug will be administered as part of this study. 输注前基线将被定义为在之前的临床研究中，仅在输注德兰异丙基莫舍帕韦克之前的时间点. 每位参与者将在先前的临床研究中输注德兰distrogene moxparvovec后进行至少5年的随访. 本研究的参与时间取决于参与者在先前的临床研究中输注德兰distrogene moxeparvovec后完成的随访时间.

This study has not yet been registered on ClinicalTrials.gov, which is currently a pre-requisite for display of detailed eligibility criteria.

• If you need assistance with ClinicalTrials.gov registration for an oncology study, please contact the Jonsson Comprehensive Cancer Center's Office of Regulatory Compliance.
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This study has not yet been registered on ClinicalTrials.gov, which is currently a pre-requisite for display of detailed eligibility criteria.

• If you need assistance with ClinicalTrials.gov registration for an oncology study, please contact the Jonsson Comprehensive Cancer Center's Office of Regulatory Compliance.
• If you need assistance with ClinicalTrials.gov registration for a non-oncology study, please contact the Office of Regulatory Affairs.

这是一项3期研究，在减少蛋白尿的基础上，评估每周两次皮下(SC)剂量pegcetacoplan与安慰剂相比对C3肾小球病变(C3G)或免疫复合物膜增殖性肾小球肾炎(IC-MPGN)患者的疗效和安全性.

Regeneron is conducting a study of an investigational new drug called DB-OTO. DB-OTO是一种正在开发的基因疗法，用于治疗由于otoferlin基因改变而导致听力损失的儿童.

The purpose of this study is to:

• Learn about the safety of DB-OTO
• Determine how well DB-OTO is tolerated (does not cause ongoing discomfort)
• Evaluate the efficacy of DB-OTO (how well DB-OTO works)

The LYNX study is a 2-part, multicenter, Phase 2 study of safety, 杜氏肌营养不良症儿童的药代动力学和生物标志物研究, double-blind, placebo-controlled part A, followed by an open-label part B.

This study has not yet been registered on ClinicalTrials.gov, which is currently a pre-requisite for display of detailed eligibility criteria.

• If you need assistance with ClinicalTrials.gov registration for an oncology study, please contact the Jonsson Comprehensive Cancer Center's Office of Regulatory Compliance.
• If you need assistance with ClinicalTrials.gov registration for a non-oncology study, please contact the Office of Regulatory Affairs.

本研究旨在比较vericiguat与安慰剂从基线到第16周对n端脑利钠肽前体(NTproBNP)变化的疗效. The primary hypothesis is Vericiguat is superior to placebo in reducing NT-proBNP at Week 16.

The primary objectives of the study are to evaluate the safety and tolerability, and pharmacokinetics (PK) of sotatercept over 24 weeks of treatment in children ≥1 to <18 years of age with PAH World Health Organization (WHO) Group 1 on standard of care (SoC). There is no formal hypothesis.

A5418 is a randomized, placebo-controlled, 开展双盲研究，以确定替可维林对实验室确诊或推定HMPXV疾病患者的治疗效果.

主要目的是评估DTX301在消除饮食蛋白限制和替代途径药物的情况下，通过维持安全的血浆氨水平来改善鸟氨酸转甲氨基酰基酶(OTC)功能的功效.

This is a 2-part, prospective, open-label, single arm, multicenter study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PDx), lenolisib在至少15例(4 - 11岁)患有活化磷酸肌肽3-激酶δ (PI3Kδ)综合征(APDS)的儿童患者中的疗效.