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A Medical Chart Review of Patients With X-Linked Myotubular Myopathy (XLMTM)

This retrospective medical chart review (RECENSUS) of approximately 100 XLMTM patients (with a goal...

A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)

This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBU...

A Multi-Center Group to Study Acute Liver Failure in Children

The PALF study group began with 20 sites and now continues with 12 sites (11 in the United States an...

A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)

The objectives of this program are: to characterize and describe the Mucopolysaccharidosis IV type A...

A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia (ACH)

This is a Phase 2, open-label, sequential cohort dose-escalation study of BMN 111 in children with a...

A Prospective Study of Remestemcel-L, Ex-vivo Cultured Adult Human Mesenchymal Stromal Cells, for the Treatment of Pediatric Patients Who Have Failed to Respond to Steroid Treatment for Acute GVHD

The study plans to treat at least 60 pediatric subjects, male and female, between the ages of 2 mont...

A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With lvacaftor

This study is designed to evaluate the efficacy and safety of VX-661 in combination with ivacaftor....

A Study Comparing Two Carboplatin Containing Regimens for Children and Young Adults With Previously Untreated Low Grade Glioma

This study is trying to learn and understand if the chemotherapy drug called carboplatin works as we...

A Study of INFUSE Bone Graft (BMP-2) in the Treatment of Tibial Pseudarthrosis in Neurofibromatosis Type 1 (NF1) (NF107-BMP2)

The current study proposes adding BMP-2 (INFUSE), an anabolic agent, at the surgical site of TPA rep...

A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy (NURTURE)

The primary objective of the study is to examine the efficacy of multiple doses of Nusinersen admini...

A Study Of Panobinostat In Children With Refractory Hematologic Malignancies

This study is for patients with relapsed or refractory Acute Lymphoblastic Leukemia (ALL), Acute Mye...

A Study of Ramucirumab (LY3009806) in Children With Refractory Solid Tumors

The main purpose of this study is to evaluate the safety of the study drug known as ramucirumab in c...

A Study With SAGE-547 for Super-Refractory Status Epilepticus

This is a randomized, double-blind, placebo-controlled trial, designed to evaluate the efficacy and...

A Two-Part Multicenter Prospective Longitudinal Study of CFTR-dependent Disease Profiling in Cystic Fibrosis (PROSPECT)

Identify and validate biomarkers that might reflect partial restoration of CFTR function and can be...

Abatacept as GVHD Prophylaxis Phase 2

This is a phase II multi-center, randomized, double blind, placebo-controlled trial. The investig...

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