A Novel, Regulated Gene Therapy (NGN-401) Study for Female Children With Rett Syndrome Read more about A Novel, Regulated Gene Therapy (NGN-401) Study for Female Children With Rett Syndrome
VE303 for Prevention of Recurrent Clostridioides Difficile Infection Read more about VE303 for Prevention of Recurrent Clostridioides Difficile Infection
LCH-IV, International Collaborative Treatment Protocol for Children and Adolescents With Langerhans Cell Histiocytosis Read more about LCH-IV, International Collaborative Treatment Protocol for Children and Adolescents With Langerhans Cell Histiocytosis
A Study to Evaluate Mavacamten in Adolescents With Symptomatic Obstructive Hypertrophic Cardiomyopathy Read more about A Study to Evaluate Mavacamten in Adolescents With Symptomatic Obstructive Hypertrophic Cardiomyopathy
Engagement in CHildhood-Onset Systemic Lupus Read more about Engagement in CHildhood-Onset Systemic Lupus
Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome Read more about Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome
A Study to Evaluate the Efficacy and Safety of Subcutaneous Amlitelimab Monotherapy Compared With Placebo in Participants Aged 12 Years and Older With Moderate-to-severe Atopic Dermatitis (COAST 2) Read more about A Study to Evaluate the Efficacy and Safety of Subcutaneous Amlitelimab Monotherapy Compared With Placebo in Participants Aged 12 Years and Older With Moderate-to-severe Atopic Dermatitis (COAST 2)
Lysosomal Acid Lipase (LAL) Deficiency Registry Read more about Lysosomal Acid Lipase (LAL) Deficiency Registry
Multi-Center Molecular Diagnosis and Host Response of Respiratory Viral Infections in Pediatric Transplant Recipients Read more about Multi-Center Molecular Diagnosis and Host Response of Respiratory Viral Infections in Pediatric Transplant Recipients
A Study to Compare Treatment With the Drug Selumetinib Alone Versus Selumetinib and Vinblastine in Patients With Recurrent or Progressive Low-Grade Glioma Read more about A Study to Compare Treatment With the Drug Selumetinib Alone Versus Selumetinib and Vinblastine in Patients With Recurrent or Progressive Low-Grade Glioma