Research

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Funding | Clinical Trials Grants

The goal of the Mooney Family Initiative for Translational and Clinical Studies in Rare Diseases award (TCSRD) is to assist investigators in the execution of their early phase human trials (including in particular first in human and pilot/feasibility studies), to allow investigators to more rapidly pursue their research which could ultimately impact the lives of pediatric patients suffering from serious or life-threatening diseases. By providing extraordinary clinical trial support to an investigator, this program seeks to enhance the translation of discovery science such that this science can have a more immediate global impact in rare pediatric diseases. The Translational Research Program (TRP) has created this new initiative to provide support to investigators in three spheres:

  1. Clinical trial costs on selected, peer-reviewed protocols.
  2. Clinical care costs for patients participating in selected clinical trials who do not have insurance coverage and no means to pay.
  3. Transportation and housing cost for families outside of New England, including international patients.

Mooney Family Initiative for Translational and Clinical Studies in Rare Diseases Awardees (TCSRD)

2016

Denise Adams - Hematology/Oncology
A Randomized Phase 2 Study of Vincristine versus Sirolimus to treat High Risk Kaposiform Hemangioendothelioma (KHE)

Monica Kleinbahn and Leslie Gordon - Anesthesia
Phase I/II Trial of Everolimus in Combination with Lonafarnib in Progeria

Sunset Agarwal - Hematology/Oncology
Radiation and alkylator-free bone marrow transplant for patients with short telomere syndromes

Janet Soul - Neurology
Pilot trial of bumetanide for newborn seizures

2015

Jennifer Whangbo - Hematology/Oncology
A Phase I Study of Individual Adult and Pediatric Patient Dose-escalated Interleukin-2 Therapy for Refractory Chronic Graft-versus-Host-Disease.”

Suneet Agarwal - Hematology/Oncology
Radiation and alkylator-free bone marrow transplant for patients with short telomere syndromes.”

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