International Dwarfism Leadership Summit report

The first International Dwarfism Leadership Summit took place in Berlin Oct. 15-16, 2016.

There were representatives from 25 patient advocacy organisations from all over the world. The global purpose was achieved, with the exception of African continent. The original purpose and focus of the meeting was to discuss global issues of concern of dwarfism support organizations around the world. Little people of America was the organiser of the summit, being the main contact Joanna Campbell, that welcomed us very well.

An image of the audience during the summit

In the first day, the morning session was devoted to the presentation of each organisation that attended the summit. We did a presentation of our work disclosing our priorities: our focus in science, our collaborative actions with clinical research, the therapies applied to children, the ALPE clinic, our international network of health professional specialists, the social empowerment strategy and much more. Here is ALPE presentation.

ALPE core actions were easily distinguishable from the other groups present, based in the points just mentioned, once most groups focused in advocacy. There was a burning theme of the designation of the groups. Ones have “little people” in their names, other “Short stature/people”, “small people”. There is no uniformization and no conclusing was taken of how to put all in the same direction in this matter.

In the second day, the main theme was the medical discussion, with a presentations of Dr. Julie Hoover-Fong (MD PhD, Director, Greenberg Center for Skeletal Dysplasias, Associate Professor of Pediatrics, Baltimore) on the overview of skeletal dysplasias. This theme was followed by a presentation of Dr. William Mackenzie, (MD, Chairman, Department of Orthopedic Surgery, pediatric orthopedic surgeon at the Nemours/Alfred I. duPont Hospital for Children and a specialist consultant in ALPE) on surgical management of bone dysplasias.

Dr. Julie is conducting an ongoing observational study for achondroplasia that can be very relevant to review the natural history of this genetic condition.

From left to right: Dr. Mackenzie, Andrea Fraschina (ACONAR- Argentina), Carmen Alonso, Inês Alves and Dr. Julie Hoover-Fong

Dr. Julie gave a second presentation on emergent treatments, but once most of the organisations presents at the summit wish not to have an approach of medical treatments for specific conditions as achondroplasia, her presentation was very basic, quick, with no remarks and without questions about the products in line now, as vosoritide. Although in the program, this was mostly a no theme.

The summit ended with no particular remarks, with few relevant questions being addressed. But it was overall, a nice weekend with time for new acquaintances.