UPDATE 6/7/2018 – NOW ACCEPTING PATIENTS
Families can contact Dr. Christina Miyake at email@example.com for further information
We received word at this past Sunday’s TANGO2 Research Foundation board meeting that the Baylor IRB protocol for a TANGO2 Study has been approved! The official title of the IRB is Understanding the underlying mechanism of TANGO2 disease – establishing Biorepository and Patient Registry.
Dr. Seema Lalani, who sits on the TANGO2 Research Foundation board, will be the Principal Investigator of the study and will be joined by Co-Investigators Dr. Lindsay Burrage, Dr. Mahshid Azamian and Dr. Christina Miyake. The work will be conducted at Baylor College of Medicine (BCM) and Texas Children’s Hospital (TCH) and will be funded by the Baylor College of Medicine. The stated objectives of the study are:
- To establish a biorepository of individuals with TANGO2-related disease to support future scientific research (blood & skin)
- To establish a comprehensive clinical database of individuals with TANGO2 disease (natural history).
- Identify patients suspected to have TANGO2 disease through Texas Children’s Hospital
It’s hard to overstate what approval of this IRB protocol means for children affected by TANGO2 and their families. The establishment of a biorepository and collection of natural histories on our children sets the stage not only for initial insights and research at Baylor and Texas Children’s – but future research projects anywhere in the world. These foundational research building-blocks, the biorepository and natural histories, will be provided to ANY qualified investigators who are studying the molecular basis of TANGO2-disease!
Here is an excerpt from the Background Information included in the IRB Protocol Report that really does a great job at summarizing the need for this type of project/study:
There is an urgent need for comprehensive understanding of the disease including pathogenesis, triggers that initiate crises in children, and preventative measures to stabilize cardiac function and skeletal muscle disease. Understanding disease mechanism requires studying disease cells/samples and thus establishing a biorepository of tissues (blood and fibroblasts) of individuals affected with TANGO2 disease is paramount. This study would allow investigators to understand the disease mechanism and in time, identify drug targets that could impact the health of these individuals, reducing significant morbidity and mortality that is currently associated with this disease.
The TANGO2 Research Foundation expresses its sincerest thanks to all those involved in making this TANGO2 IRB protocol a reality at the Baylor College of Medicine and Texas Children’s Hospital with a special thanks to Drs. Lalani, Burrage, Azamian and Miyake. Your caring and dedication to your patients and profession are an inspiration to us all.
CONTACT INFORMATION AND DETAILS FOR PARTICIPATING IN THE STUDY WILL BE POSTED SHORTLY