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At Cure 4 the Kids Foundation, we recognize that follow-up care is essential to the health and well-being of childhood cancer survivors. Through our Long-Term Follow-Up Program (LTFU), we provide a comprehensive assessment, educational resources, and a treatment summary specific to each individual survivor.
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Cure 4 The Kids by Admin - 5M ago
FORE! Join us at Red Rock Country Club’s Private Mountain Course for an incredible morning of golf while helping support children with cancer, rare diseases and inherited bleeding disorders.   Register Here
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We are happy to recognize that there have been great improvements in caring for people with sickle cell disease (SCD); however, there are still some things we don’t know about the life-long condition which can have devastating effects on a patient’s health. Even the exact number of those affected by SCD in the United States
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Sickle Cell   Protocol:D5136C00009   Condition or Disease: Sickle Cell Disease   Study Type: Interventional (Clinical Trial)   Status: Open for enrollment   Study Title: A randomised, double-blind, parallel-group, multicenter, phase III study to evaluate the effect of ticagrelor bid versus placebo in reducing the number of VOCs in paediatric patients with sickle cell disease.
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Genetics   Protocol:VTS-270-303   Condition or Disease: Niemann-Pick Disease, Type C   Study Type: Interventional (Clinical Trial)   Status: Opening Mid 2019   Study Title: An Open-label, Multicenter Safety and Tolerability Study of VTS-270 (2-hydroxypropyl-ë_-cyclodextrin) in Pediatric Subjects Aged < 4 Years With Neurologic Manifestations of Niemann-Pick Type C (NPC) Disease     Principal I
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Genetics   Protocol:VTS-270-001   Condition or Disease: Niemann-Pick Disease, Type C   Study Type: Expanded Access   Status: Open for enrollment   Study Title: An Open-label Expanded Access Treatment Protocol for VTS-270 (2-hydroxypropyl-ë_-cyclodextrin) in Patients With Neurologic Manifestations of Niemann-Pick Type C Disease (NPC)     Principal I investigator: Alan Ikeda, MD     More
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Genetics   Protocol:VTS-270 IND   Condition or Disease: Niemann-Pick Disease, Type C   Study Type: Expanded Access   Status: Open for enrollment   Study Title: Intrathecal 2-Hydroxypropyl-ë_-cyclodextrin for Neurological Decline in Patients with Niemann-Pick Disease Type C1     Principal I investigator: Alan Ikeda, MD
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Sickle Cell   Protocol:GBT440-034   Condition or Disease: Sickle Cell Disease   Study Type: Interventional (Clinical Trial)   Status: Open for enrollment   Study Title: An Open Label Extension Study GBT440 administered Orally to Patients with Sickle Cell Disease Who Have Participate in GBR440 Clinical Trials.     Principal I investigator: Alan Ikeda, MD  
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Sickle Cell   Protocol:GBT440-031   Condition or Disease: Sickle Cell Disease   Study Type: Interventional (Clinical Trial)   Status: Enrollment Paused   Study Title: A Double-blind, Randomized, Placebo-controlled, Multicenter Study of GBT440 Administered Orally to Subjects with Sickle Cell Disease     Principal I investigator: Alan Ikeda, MD     More Info: https://clinicaltrials.gov/ct2/show/NCT03036813  
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Genetics   Protocol:110-504   Condition or Disease: Mucopolysaccharidosis Type IVA   Study Type: Observational   Status: Open for enrollment   Study Title: Morquio A Registry Study (MARS)     Principal I investigator: Angela Berg, DNP, APRN, CPN
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