Search Results

• A RETROSPECTIVE CONTINUATION STUDY IN SMALL FIBER NEUROPATHY IN CHILDREN: PRESENTATION & CLINICAL FEATURES

  A RETROSPECTIVE CONTINUATION STUDY IN SMALL FIBER NEUROPATHY IN CHILDREN: PRESENTATION & CLINICAL FEATURES

• A Multicenter, 12-Month, Randomized, Double Blind, Placebo-Controlled Phase 3 Efficacy and Safety Study of Daily Oral LUM-201 in Naïve-to-Treatment, Prepubertal Children with Growth Hormone Deficiency

  A Multicenter, 12-Month, Randomized, Double Blind, Placebo-Controlled Phase 3 Efficacy and Safety Study of Daily Oral LUM-201 in Naïve-to-Treatment, Prepubertal Children with Growth Hormone Deficiency (GHD)

• A Study Testing the Combo of Dasatinib or Imatinib to Chemo Treatment W/ Blinatumomab for Children, Adolescents, & YA W/ Ph+ or Ph-Like B-cell B-ALL

  A Study Testing the Combination of Dasatinib or Imatinib to Chemotherapy Treatment With Blinatumomab for Children, Adolescents, and Young Adults With Philadelphia Chromosome Positive (Ph+) or ABL-Class Philadelphia Chromosome-Like (Ph-Like) B-cell Acute Lymphoblastic Leukemia (B-ALL)

• A 4-Week, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Assess the Safety and Efficacy of Tenapanor for the Treatment of Irritable Bowel Syndrome with Constipation (IBS-C) in Pediatric Patients 6 to Less than 12 Years Old

  A 4-Week, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Assess the Safety and Efficacy of Tenapanor for the Treatment of Irritable Bowel Syndrome with Constipation (IBS-C) in Pediatric Patients 6 to Less than 12 Years Old

• Molecular and Clinical Risk-Directed Therapy for Infants and Young Children With Newly Diagnosed Medulloblastoma

  Phase 2 Study of Molecular and Clinical Risk-Directed Therapy for Infants and Young Children With Newly Diagnosed Medulloblastoma

• Endoscopic versus Shunt Treatment of Hydrocephalus in Infants (ESTHI)

  Endoscopic versus Shunt Treatment of Hydrocephalus in Infants (ESTHI)

• A 12-Week, Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study to Assess the Safety and Efficacy of Tenapanor for the Treatment of IBS-C in Pediatric Patients 12 to 18 Years Old

  A 12-Week, Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study to Assess the Safety and Efficacy of Tenapanor for the Treatment of Irritable

• LONG-TERM STUDY OF TRIPTORELIN IN GIRLS WITH CENTRAL PRECOCIOUS PUBERTY

  LONG-TERM STUDY OF TRIPTORELIN IN GIRLS WITH CENTRAL PRECOCIOUS PUBERTY

• DAY101 vs. Standard of Care Chemotherapy in Pediatric Patients With Low-Grade Glioma Requiring First-Line Systemic Therapy (LOGGIC/FIREFLY-2)

  LOGGIC/FIREFLY-2: A Phase 3, Randomized, International Multicenter Trial of DAY101 Monotherapy Versus Standard of Care Chemotherapy in Patients With Pediatric Low-Grade Glioma Harboring an Activating RAF Alteration Requiring First-Line Systemic Therapy

• A Phase 2 Study of Blinatumomab in Combination With Chemotherapy for Infants With Newly Diagnosed Acute Lymphoblastic Leukemia With Randomization of KMT2A-Rearranged Patients to Addition of Venetoclax

  A Phase 2 Study of Blinatumomab in Combination with Chemotherapy for Infants with Newly Diagnosed Acute Lymphoblastic Leukemia with Randomization of