Summary of the Conference	Video of Conference

Video of Families that Attended	Video of Children Thanking Sigma Tau

Slideshow of Families that attended	Reflections of the Euro Conference: By Claudia Brugger	ENGLISH OR GERMAN

I want to thank Steve Broome for help with the Video Streaming and Bruce Mund for the Summary.

CONFERENCE SUMMARY
130 PEOPLE FROM NINE COUNTRIES ATTENDED
Click any of the pictures to view them enlarged.

This Conference was made possible because of very generous funding provided by Sigma Tau Pharmaceuticals, Inc. We owe them our heartfelt thanks for this event!

What a pleasant surprise to find Bergamo...A very busy city with a population of 122,000. With heavy traffic, many Cathedrals, bells ringing about every hour....Very Beautiful!!........The old city "CITTA ALTA" in the hills with its cobblestone streets and ancient buildings, many restaurants, plaza, church steeple, stone walls and arches, made a most pcturesque back drop from our hotel rooms.

Our first formal meeting started on the morning of September 4, 2000. In the auditorium of Villa Camozzi, families from nine countries were in attendance, parents, children, doctors, and representatives of drug companies and research centers as well as Cystinosis Foundation volunteers. Two young Italians from Milano, Roberto Guerrino and Cristina Bonani provided simultaneous translations, mostly English to Italian, but also when needed, Italain to English.

Jean Hobbs-Hotz, President introduced Carmela Lotrecchiano, Laura Chichilo, and Gaetano DiBenedetto, from the New Jersey Cystinosis Foundation, New York Chapter,

United States. No interpreter was needed as they all spoke Italian and English fluently. Cystinosis claimed the life of Gerardo Lotrecchiano, son of Carmela and Jerry Lotrecchiano, when he was five years of age. His life was the inspiration for their forming the Cystinosis Foundation, New Jersey. Carmela and Jerry have an adult daughter Gerardina, who has cystinosis and attended the Conference. Gerardina is a gifted dancer. Laura is an older sibling and is very involved with fundraising events which contributes to research projects. Gaetano DiBenedetto, President of C.F., N.J. has been a guiding light since the inception of the organization.

Karen Ritchie gave a workshop on "The Grieving Process",

entitled "Shattered Dreams, the Parents' Struggle with Childhood Disability." In her presentation the 6 stages of grieving /loss were discussed. She stressed the importance of experiencing the stages of Denial, Anger, Guilt, Depression, Anxiety, and Fear in your own time and your own way, in order to cope with the "losses" in your lives. the grieving process is both necessary and inevitable to a healthy life. Joshua Hotz topic was "Walking in My Shoes."

Josh is a teenager with cystinosis. He spoke briefly of his living with cystinosis. Don Hammond spoke on "Getting the Most out of Life While Living With a Chronic Disorder." Don has assisted at several Cystinosis Foundation Conferences, always sharing his wisdom and strength with families

Dr. Roberto Dall'Amico of the University of Padua, Italy told us of a study of 46 Cystinotics in Italy using Growth Hormone, and that 25 out of 44 Italians with cystinosis take the eyedrops.

Dr. Angela Ballantyne from Dr. Trauner's neurology lab in U.C. San Diego, explained for us again the findings that Cystinotic children learn best by hearing. Dr. Minnie Sarwal of Stanford University initiated the only steriod avoidance immunosuppressive protocol in the United States, 18 mos ago (Click Here to see the video)

with Dr. Oscar Salvatierri, the renowned transplant surgeon. She said "There have been no acute rejections, no cholesterol problems, the graft function is better, and children are growing much better."

The families in attendance spoke to the audience

of their family history with Cystinosis (also,see Conference video) and the Shakar family from Israel presented the progress of their son, Ohad, Age 10 1/2. Dr. Georg Wolff,

Clincial Psychologist from the Children's Clinic in Hannover, Germany, discussed the fact that an adolescent has a high degree of changes, mood swings, and extremes. His concluding thought...."Your kids are lucky to have parents like you." Dr. Corrine Antignac of the Children's Hospital in Paris, France spoke about the French Collaborative study of August 1999, proving that Cystinosis involves the Lysosomal Membrane.

Dr. Gianfranco Rizzoni from the Children's Hospital in Rome, reported on the Wuehl Study of 1999 using 36 patients on Growth Hormone, over a period of one year improved growth was shown without accelerated deterioration of Kidney function. Another study with 18 children in Rome showed the biggest growth in first 3 years of life. Dr. William Van't Hoff from Guy's Hospital in London, England reported on studies to explore how cells work with Cystinosis. What detrimental effects might "Cystagon" have on the Cell? Should we be looking for a

Time-Release capsule for "Cystagon"? Professor Silvio Garattini, M.D. director of the Mario Negri Institute, Milan, Italy related to us that in 1993 the US passed the "Orphan Drug Law" permitting special licensing and financial benefits to companies developing rare or "Orphan Drugs", and in December 1999, Europe passed a similar law.

Dr. Jess Thoene, of Tulane University, U.S. showed slides of Cystine Crystals as seen under a microscope; explained how the cysteamine acts as a porter (Carrier) to carry Cystine out of the cell. Currently funded by the Cystinosis Foundation, Dr. Thoene's lab is working on producing Knock Out Mice. The Mouse has 80% homology with man. Dr. Doris Trauner of the University of California, San Diego,

Department of Sciences related that there are crytstals in the brain of cystinotics. It is not known how or why they are present. Dr. Trauner and Dr. Ballantyne spent much of their time at Ranica testing the children with Cystinosis. Testing was voluntary and the families have received the results of the testing.

Dr. Michel Broyer of the Children's Hospital in Paris, France, illustrated the dosage for eyedrops as 0.5 percent cysteamine 4 to 6 times daily.

The oral dosage as 10 to 50 or 60 milligrams per kilogram of body weight every 6 hours. One study followed 44 patients and showed the benefits of cysteamine. Dr. William Gunnarsson, President of Orphan Europe, Paris, explained that the Federal Drug Administration and European Board of 15

countries and Japan collaborate on new drugs. Jonathan Terry, Founder of the Cystinosis Foundation of the United Kingdom and Ireland, spoke of taking an early retirement to spend the necessary time to run a foundation. Dr. Erik Harms, from the Children's Hospital at the University of Muenster, Germany charted cysteamine progress in Germany, he stressed the early diagnosis of cystinosis and treament with cysteamine.

Dr. William Gahl, of the National Institutes of Health, U.S.,

showed slides of the needle-like crystals and protrusions of the eye membrane in the untreated eye. In Type I and II Cystinosis the crystals in the eye are gone in 6 to 24 months by taking eyedrops 8 to 12 times a day.

Heather Kirby, Manager of Special Products, Mylan Pharmaceuticals, Inc., discussed the development of Cystagon, Mylan developed cysteamine from liquid form into Cystagon, a capsule, and they sheperd the process until obtaining FDA approval in 1994.

Dr. Edward Lemanowicz,

of Sigma Tau Pharmaceuticals, Inc., related to us that Sigma Tau also markets Carnitor, another Orphan Drug, Offices of Sigma Tau are in Rome and Maryland, Pharchemia in Bergamo supplies cysteamine. Sigma Tau's Eyedrops will be launched commercially in 2002 under the name "CYSTAVISION", these eyedrops will be stable for 7 mos; 12 mos stable at room temperature unopened; 24 mos stable unopened if refrigerated; and 7 days stable at room termperature as an open bottle.

Dr. Jerry Schnieder of UC San Diego Medical School (Dean of Academic Affairs), has devoted most of his medical career to the diagnosis, care and treatment of Cystinosis. He related that the cystinosis foundation started in 1982; Cysteamine approved in 1994. Dr. John McCormick, Deputy Director, Office of Rare Disease Products, Federal Drug Administration explained that an Orphan Drug earns 7 years exclusive rights for the company developing it. Mr. Anders Olauson, Chairman of Eurodis, Director of Agrenska, Center for Rare Disorders, Sweden (Headquartered in Paris), related that 1,908 disabled children have been through the center. In 1997 Eurodis started in order to include Europe in rare disorders.

Karen Gledhill from New York, U.S. has late onset cystinosis, told us she had a transplant in 1983, was under the care of Dr. Schneider orignially, and took Cyclosporine experimentally and has had the kidney for 19 years, and is doing very well. Mara Fogliada, from Gordona, Italy is working to found a Cystinosis Foundation, Italy. Mara successfully contacted and organized the Italian families who attended the Conference. She coordinated the translators and worked in a number of capacities.

While most adults were meeting

these 3 very busy days, the children were being tested but also being involved in interesting fun activities under the care of Don Hammond (San Jose, Calif) a family counselor, and "Pied Piper" for children.

THANK YOU TO ALL OF OUR VOLUNTEERS,
OUR MOST GENEROUS SUPPORTERS, AND THE SPEAKERS

The International Cystinosis Foundation Conference was a united global effort by the Cystinosis Foundation to bring the Cystinosis Community together. Sigma-Tau, provided the funding which made the Conference possible. The global volunteers donated much of their time and talents, for their love of families and individuals with cystinosis.
Our sincere gratitude to Frank Ritchie, Carl Schleuder,, E. David Hotz, Velyna Morales, Karen Gledhill, Sue Stojanovich, Bruce Mund, Karen Ritchie, and Jean Hobbs Hotz of the United States; from England, Jonathan Terry, and Brian Long; Germany, Claudia Brugger; France, Francois Couppey. Especially we want to render thanks to William Gunnarsson for his advice and council. We are grateful for the presence of Justina Lambert Deeds, Corporate Affairs Director, and Gianofranco Fornasini, Ph.D., Director of Regulatory Sciences, from Sigma-Tau. (See Video "Children")

We are extremely grateful to the following doctors, researchers, and speakers for their continued compassion, dedication, and work which have provided better treatments, understanding, and comfort to cystinosis patients and their families.

Euro Conference 2000