The 7th International Cystinosis Conference, Roissy,
France
"BEYOND BORDERS"
The 7th International Cystinosis Conference was held in Roissy, France, June 28 - July 1, 2012. Roissy is a beautiful little city just outside of Paris, France. Just a few minutes from Charles De Gaulle airport.
The conference hosted 265 attendees and the Roissy Marriott was the venue for the conference,
it is a
beautiful hotel, with a very nice area for the attendee's to sit and
socialize with each other. The Marriott provided free transportation to
and from
the airport and train station for those that wanted to visit Paris.
The
over 40 children in attendance enjoyed a variety of activities during
the conference that were planned by Laetitia Lepoix, president of
Cystinoise and Francois Couppey, AIRG France. Francois Couppey worked very
hard to secure FREE tickets to Euro Disney for the children and adult
escorts. The children's breakout room had many things for them to do,
including a lady that entertained the kids making balloons, and across
the street was a beautiful park with climbing equipment and slides that
they played on.
Thursday
evening was the Welcome Reception, and for those that arrived early,
we shared beer, wine, soft drinks, and snacks. Everyone
mingled. Those families that have been to previous conferences renewed
old friendships, those that were new to the conference were meeting
and making new friends. There was plenty of entertainment, and
of course the kids became fast friends and took over the hotel.
We want to thank Raptor for the fabulous Welcome Reception.
Friday morning started with a warm welcome from Patrick Niaudet, Scientific Advisor; Daniel Renault, President AIRG, and Valerie Hotz, Executive Director, Cystinosis Foundation, Inc. Jerry Braza was the keynote speaker. He elaborated on the value of developing a positive outlook while coping with cystinosis and the importance of building good relationships that help sustain us." We were the first to see the newest professionally created video of The Cystinosis Foundation's Celebrity Spokesperson, Abby Riggs Over the next two and a half days we heard from speakers from all over the world: Dr. Langman, Dr. Gahl, Dr. Trauner, Dr. Del Monte, and Dr. Dohil, USA; Dr. Antignac, Dr. Cherqui, Dr. Cochat, Dr. Servais, Dr. Deschenes, and Dr. Niaudet, France; Dr. Levtchenko, Belgium; Dr. Topaloglu, Turkey; Dr. Emma, Italy; Dr. Webb, U.K. Betty Cabrera, from the University of California, San Diego, where she coordinates Cystinosis clinical trials, and as curator of the "Cure Cystinosis International Registry" (a collaboration between The Cystinosis Foundation and the Cystinosis Research Foundation. If you have not registered with the CCIR, we encourage you to do so by clicking on this link. The kids received a free trip to Disneyland Paris, Francois Couppey worked with Disneyland Paris to provide and afternoon at the park.
Afternoon
breakout session included a workshop for Grandparents and extended
family and a patient panel discussing "Living With Cystinosis".
Feedback was excellent and participants were many. In
the Plenary Sessions, topics ranged from "50 Years of Cystinosis
Research" to an Announcement by Dr. Cherqui the Bone Marrow/Gene
Therapy study is about to begin. Just a note: The
Cystinosis Foundation, Inc. here in the USA, was the first to
recognize the work being done in France. The Foundation
approved funding to Dr. Antignac at Necker Hospital in Paris,
France. Dr. Antiqnac's group identified the gene involved in
Cystinosis, the CTNS gene. Dr. Cherqui assisted with discovery of
the gene, and brought that discovery to the U.S. along with the
Cystinosis mouse, and started the project "Stem cells and gene
therapy for cystinosis" at the Scripps Research Institute. She
has demonstrated that transplanting bone marrow stem cells leads to the
long-term preservation of the kidney in Ctns
mice.
Throughout Saturday the most knowledgeable doctors and researchers came to the podium to speak on an array
of topics important to the families and patients attending the
conference. Dr. Trauner spoke on "Muscle Wasting" and
"Neurological complications in cystinosis". Dr. Dohil spoke
on "Gastro-intestinal problems. Dr. Niaudet discussed :Renal
Transplatation". Dr. Del Monte talk to us about "Ocular
manifestations in cystinosis", and "New cysteamine collyriums".
We were updated on Cystadrops, Sigma-Tau is just waiting for FDA
approval of manufacturing to provided patients in the U.S. with the eye drops. There were a number of workshops breakout sessions
Children\Parents, adults, and young adult patients.
Saturday
evening, Cystinose France was gracious enough to provide the
entertainment, a band named "ECHOES" played for all of the attendees and
everyone had a wonderful time listening and dancing to the sounds of
"ECHOES" a partner of Cystinose France. During the conference
guests were invited to share their thoughts and hopes for the
future. This project culminated in the production of the short
video by Raptor, entitled "My View", which Raptor generously donated
to the Cystinosis Foundation. We all enjoyed watching the
video on Saturday night. As the band ECHOES played, a conga
line of parents and children danced through the room and across the
foyer with much laughter.
Sunday was a half a day of Question and Answer sessions on topics
like "Neurological and Psychological problems" (Dr. Trauner);
"Gastro-intestinal problems, feeding and growing" (Dr. Dohil);
"Preserving renal function" (Dr. Langman). The conference was
concluded by noon, with closing remarks from Dr. Niaudet, Daniel
Renault, and Karen Ritchie (Board Member, Cystinosis Foundation,
Inc.) expressing
appreciation to all of the Speakers, Attendees, and to all of our
partners, that helped to make this one of the most successful
International Conferences we have had.
The Cystinosis Foundation, Inc., would like to thank our partners for being committed to our community and for helping us to bring families, doctors, researchers, and professionals from all over the world together. For supporting this conference and helping us to make it one of the most successful and highly attended Conferences in the nearly 30 years that we have brought support to the families and patients with Cystinosis.
THANK YOU!
AIRG France; Cystinose France; Raptor Pharmaceuticals; Orphan Europe; Sigma-Tau Pharmaceuticals;
