Congenital Hyperinsulinism International

December 2018 Newsletter

CHI Newsletter 

December 2018

Dear Friends,

As 2018 draws to a close, we want to share with you how phenomenal this year has been for the congenital hyperinsulinism (HI) community and important news. There are so many reasons for hope and optimism. Thank you for your all you do to support the work of CHI, advancing research for better treatments and cures, raising awareness of HI to prevent brain damage and death, and being there for HI families every step of
the way.

Enjoy our End of Year HIlights!

Clinical Trials

The HI community needs new therapies and approaches for patients who do
not respond to currently available treatments, and clinical trials that
safeguard the health of study participants are necessary for the development
of new HI products. There are a number of trials currently recruiting or soon
to be recruiting. Check out the clinicaltrials.gov webpage for a list of HI-related
trials here.

The HI Global Registry

CHI is celebrating the two-month anniversary of the HI Global Registry, which will generate new insights into congenital hyperinsulinism (HI), drive new research for treatments and cures, and support the success of clinical trials. We launched October 9, 2018 and already have 120 participants who have contributed invaluable knowledge and details about the experience of living with HI. 

Learn more and join the registry HERE!


CHI Family Conferences


Congenital Hyperinsulinism International (CHI) and Cook Children’s hosted the 2018 Congenital Hyperinsulinism Family Conference from July 27-29, 2018, in Fort Worth, Texas and CHI partnered with the Medical School of the National Kapodistrian University of Athens on the 2018 Athens Congenital Hyperinsulinism Family Conference in Athens, Greece, September 25-26.

230 people attended the conferences including kids with HI, their siblings, parents, physicians, researchers, and members of the biotech industry. Participants learned all about HI through presentations from specialists, panel discussions, small group interactive sessions, speed dating with the specialists, the sharing of family stories, and informal time for networking and support. Presenters and participants came from 13 countries. Another 461 people tuned in to our Facebook Live for the “Ask the Experts” sessions, expanding the reach of our conferences in a very significant way. One participant said the most meaningful part of the conference was “hearing from other families about the struggles they go through on a day-to-day basis and knowing we are not the only parents going through the stresses of living with HI.”

 

Roller Coaster Day at Luna Park

Coney Island 

Every day with HI is a Roller Coaster… but not every day with HI is ROLLER COASTER DAY! On September 15th, 2018, CHI hosted our first-ever Roller Coaster Day at Luna Park, Coney Island in Brooklyn, NY, and it was a day of pure fun for family and friends!


 

The CHI Genetic Testing Program

 

 

In partnership with the world-leading hyperinsulinism genetic research team at the University of Exeter Medical School, CHI is funding genetic testing for babies and children from around the world who could not otherwise afford the testing. Getting the right diagnosis and treatment can help avoid brain damage and other severe complications. Dr Sarah Flanagan, of the University of Exeter Medical School, who leads the research program, said: “This genetic testing is absolutely vital for families. For many children with this condition the only way to prevent brain damage is to undertake pancreatic surgery. The genetic result can help determine whether all or just part of the organ should be removed, which can mean better outcomes for the children. I’m delighted that Congenital Hyperinsulinism International is providing funding for this crucial work for people worldwide who could not otherwise afford this test.” In the first quarter of the program from July 1 to the end of August, 48 patients from 28 countries received genetic testing through this program. In 2019, about 200 patients are expected to receive this support thanks to this wonderful partnership.  

 

Million Dollar Bike Ride

 

CHI is delighted to share the Orphan Disease Center of the University of Pennsylvania has just selected Dr. Amanda Ackermann, MD, PhD of the Children’s Hospital of Philadelphia to be the grant recipient for her project: “Vitamin E Supplementation in Hyperinsulinism/Hyperammonemia Syndrome.” Dr. Ackermann will have funding for one year in the amount of $84,020. These funds are made possible by generous CHI donors, the CHI Raring to Go for CHI Team and the University of Pennsylvania which matched the funds. There were six excellent grant proposals submitted from researchers in Europe and the US.

 

ESPE Booth

 

CHI was represented at the 2018 European Society of Pediatric Endocrinology (ESPE) Meeting. With our partner advocates from Germany, Spain, and Austria, CHI raised awareness of HI and hypoglycemia and shared information about the HI Global Registry, and we had conversations with over 200 pediatric endocrinologists from all over the world. We learned of specific issues facing children with HI in many countries around the world, as well learning of the research interests of these professionals. We recruited specialists to work on HI materials in foreign languages.

 

Pediatric Endocrinology
Global Access Survey

In October, CHI developed the Pediatric Endocrinology Global Access Survey. With the contacts we have made at international meetings over the last few years, we sent out a survey to learn more about HI treatment access issues in many locales around the globe. To date, we have received 80 responses from pediatric endocrinologists in 43 countries from Africa, Asia, Europe, Australia, and North and South America. The survey remains open, and will guide us as we work with experts around to world to improve access to medication and care around the world.

2018 Sugar Soirée

CHI hosted the 2018 Sugar Soirée on November 17 at Fenway Golf Club in Scarsdale, New York. Emmy Award-winning “CBS This Morning: Saturday” co-host and CBS News national correspondent Michelle Miller hosted the event which was chaired by CHI supporters Thaïs and Marc Stuart. We honored Dr. Lisa States, pediatric radiologist at the Children’s Hospital of Philadelphia, for her work on the study FDOPA PET/CT, which has been integral to the cure many babies and children with HI have received through life-changing surgery. The Stuarts were also honored with an award for their altruism and generosity in the face of loss. Isabel Calderón, mother of an HI child, was honored for her work on patient-powered research, including the HI Global Registry. Funds raised at the Sugar Soirée will support vital awareness to prevent prolonged hypoglycemia which can lead to brain damage and death; research supporting better treatments and a cure, and support for patients including helping them receive specialized treatment, genetic testing, medication, supplies and care for families living with hyperinsulinism; and meetings and conferences for HI families and medical personnel.

 

Berlin, Germany Conference

CHI Executive Director Julie Raskin presented at the Kongenitaler Hyperinsulinsimuse.V Hyperinsulinism Family Conference in Berlin, Germany. Invited by Ulrike Seyfarth, President of Kongenitaler Hyperinsulinsimuse.V, Julie had the opportunity to learn about the HI landscape in Germany and to introduce the Berliners and others attending the conference to the HI Global Registry. The results are in: closer ties between the organizations and plans to collaborate on the 2019 CHI Family Conference in Vienna and making CHI projects more available to people whose first language is German.

“What Is Hyperinsulinism?” Posters

CHI posters, “What is Hyperinsulinism” and the “Signs and Symptoms of Hypoglycemia” are now available in 18 languages. The most recent additions are Arabic, Bulgarian, Greek, and Hebrew. The posters are a fantastic way to raise awareness of HI all over the world. We’re always looking to expand the collection, so please drop us a line if you know someone with the expertise to translate the posters into more languages.

https://congenitalhi.org/chi-posters/


 

 

 

We’re gearing up for another great year.

We cherish each and every member of our community: HI families and our sweetest volunteers and most generous donors.

Wishing you all very happy holidays.

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