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Cystinosis Research Network Family Conference July 16 18, 2015


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Cystinosis Research Network Family Conference



July 16 – 18, 2015

DoubleTree by Hilton Chicago ̶ Magnificent Mile

Chicago, Illinois
AGENDA


Detailed Draft Agenda • 5/22/15





Wednesday, July 15




Room


3:00 pm –

8:00 pm



CRN Board of Directors Meeting

Closed Session

Fairbanks

Thursday, July 16




Room


12:00 pm – 5:00 pm

Registration / Information Desk


Lasalle Ballroom Lobby




Dream, Achieve, Inspire” art exhibit

"Dream, Achieve, Inspire" an Art Exhibit Artists with cystinosis from around the world have come together to share their creativity for global cystinosis awareness. More than 80 artists from 29 countries have united to bring cystinosis struggles to light. Each piece tells a story of strength and hope. The exhibit crosses language and cultural barriers in an effort to express the need for research, better treatments and in some countries access to life saving medication where there is none. "Dream, Achieve, Inspire" is an emotional journey through the hearts of those with cystinosis...Enjoy the beautiful ride!




Lasalle Ballroom

2:00 pm –

4:00 pm


CRN Scientific Review Board Meeting

Closed Session


Fairbanks

5:00 pm –

7:00 pm



CRN Welcome Reception

Hosts: CRN Board of Directors

Complimentary

Please join us for drinks and appetizers and an opportunity to meet the families and physicians attending the conference.




Pool Deck/Lasalle Ballroom


5:00 pm – 9:00 pm

Artists in Residence

Snow City Arts invites you to participate in the creation of a mural representing our conference. All attendees are encouraged to add their own twist to the project that will become part of the Art Exhibit and travel the world to other cystinosis conferences. Be a part of our artistic love letter to the cystinosis community! The final piece will be unveiled at the closing party.




Huron

7:30 pm –

9:00 pm


Family Introductions

Pam Woodward, CRN Vice President Family Support


Lasalle Ballroom

Friday, July 17




Room


7:00 am –

8:30 am


Breakfast

Complimentary


Lasalle Ballroom

8:00 am –

5:30 pm


Registration/Information Desk

Lasalle Ballroom Lobby

8:00 am –

5:30 pm


Child Care Open

State

8:30 am –

9:00 pm


Teen Lounge Open

Room?




Dream, Achieve, Inspire” art exhibit

"Dream, Achieve, Inspire" an Art Exhibit Artists with cystinosis from around the world have come together to share their creativity for global cystinosis awareness. More than 80 artists from 29 countries have united to bring cystinosis struggles to light. Each piece tells a story of strength and hope. The exhibit crosses language and cultural barriers in an effort to express the need for research, better treatments and in some countries access to life saving medication where there is none. "Dream, Achieve, Inspire" is an emotional journey through the hearts of those with cystinosis...Enjoy the beautiful ride!




Lasalle Ballroom

9:00 am - 5:00 pm

Artists in Residence

Snow City Arts invites you to participate in the creation of a mural representing our conference. All attendees are encouraged to add their own twist to the project that will become part of the Art Exhibit and travel the world to other cystinosis conferences. Be a part of our artistic love letter to the cystinosis community! The final piece will be unveiled at the closing party.




Superior I




Global Genes Teen Adventure

Michigan

8:30 am –

8:35 am



Welcome and Opening Remarks

Jeff Larimore, CRN President


Lasalle Ballroom

8:35 am –

8:50 am



Cystinosis Research Network – Your Advocacy Group

Jeff Larimore, CRN President

Lasalle Ballroom

8:50 am –

9:35 am


Who Sets Our Limits?”

Bonner Paddock

Bonner Paddock is the Founder of the OM Foundation (OMF), which promotes the idea that any one person can make a difference if they dedicate themselves to look beyond their own personal limits to achieve their goals. Bonner is the first person with Cerebral Palsy to ascend Mt. Kilimanjaro and to finish the infamous Ironman World Championship unassisted. He has since raised over $1 Million Dollars to help build and support early learning centers in the US & Africa, which provide therapy for special needs children.  He has also recently launched his first book, One More Step, a memoir about his life living with Cerebral Palsy, conquering the incredibly challenging physical feats described above, and starting his own foundation.  Listen to Bonner's inspirational story as he shares with you what held him back for 30 years and how he pushed beyond his own limits to achieve success.  www.1man1mission.org


Lasalle Ballroom

9:40 am –

10:10 am



Cystinosis Overview

William A. Gahl, M.D., Ph.D., Clinical Director, National Human Genome Research Institute; Director, National Institutes of Health Undiagnosed Diseases Program

Nephropathic cystinosis, a lysosomal storage disorder due to defective transport of cystine out of lysosomes, results from mutations in CTNS. Almost half the patients in North America and Europe are homozygous for a 57-kb deletion in CTNS.

Without treatment, children with cystinosis suffer from renal Fanconi syndrome and its complications, growth retardation, photophobia, and end-stage renal failure requiring kidney transplantation. Treatment with oral cysteamine, which can reduce cellular cystine levels by 95%, dramatically slows glomerular deterioration and normalizes growth. Based upon our examinations of 100 adult cystinosis patients between 1985 and 2006, we found striking rates of mortality (33%; mean age 29 years) and morbidity (24-75% for each complication), specifically related to hypothyroidism, hypergonadotropic hypogonadism (in men), pulmonary insufficiency, swallowing abnormalities, myopathy, retinopathy, vascular calcifications, and diabetes. In adults, long-term (>8years) oral cysteamine therapy was associated with significantly greater height and weight, older age at renal transplant, lower serum cholesterol levels, and lower rates of morbidity and mortality. In fact, as duration of cysteamine therapy increased, the frequencies of non-renal complications of cystinosis decreased. All symptomatic cystinosis patients should receive oral cysteamine therapy, and the earlier diagnosis is made and treatment initiated, the better the prognosis. This talk will serve as background for discussions of current pursuits into cystinosis clinical and basic research.


Lasalle Ballroom

10:10 am – 10:25 am

Break

Lasalle Ballroom Lobby

10:30 am -

11:20 am


Management of Infants and Children with Cystinosis

Moderator: Craig B Langman, MD

Setting the stage

Larry Greenbaum, MD, PhD: A physiological approach to treatment of the Renal Fanconi Syndrome

Paul Goodyer, MD: Water Conservation with Indomethacin in Cystinosis

Galina Nestarova, MD: Rachitic Bone Disease

Neveen Soliman, MD, PhD: Chronic Kidney Disease – Metabolic Bone Disease

Ewa Elenberg, MD, MS: Gastrointestinal Manifestations

Questions to all from the audience




Lasalle Ballroom

11:25 am – 12:15 pm

Adolescent Issues in Cystinosis

Moderator: Elena Levtchenko, M.D. Panelists: Paul Grimm, M.D., Minnie Sarwal, M.D., Ph.D., Robert Kleta, M.D., Ph.D., Maria Helena Vaisbich, M.D.
Adolescence in cystinotic patients is associated with different matters related to the development of the systemic disease in addition to the renal disease, the progression to ESRD and the need for renal replacement therapy, and the transition to adult age and adult clinics. During this session the experts will focus on the specific issues of endocrine involvement and pubertal development in cystinosis, whether the management of renal transplant in cystinosis differs from other kidney diseases and how adolescent cystinosis patients can be better prepared to enter the adult world, including disease acceptance, the appropriate studies and job choice, medication compliance and quality of life.
Short presentation by the experts will be followed by Panel discussion.


Lasalle Ballroom

12:15 pm – 1:15 pm

Luncheon

Complementary

Lasalle Ballroom

1:15 pm – 2:15 pm

Poster Session

This session will showcase a mix of science, medicine, industry, advocacy group and patient experiences to provide an interactive experience for both family and professional attendees. Researchers, clinicians, industry, advocacy representatives, students, patients, and caregivers will be invited to exhibit their latest research findings, treatment breakthroughs, advocacy group updates and real patient and family experiences. This will be an interactive session where exhibitors will be available to discuss their work or experiences with those attending. We invite you to browse the posters and take this opportunity to ask the authors and presenters questions.




Huron

2:20 pm – 3:10 pm

Transition from Pediatric to Adult Care

Moderator: Frederick Kaskel, M.D., Ph.D. Panelists: Richard Simon, M.D., Patrick Gipson, M.D., Jess Thoene, M.D., Robert Kleta, M.D., Ph.D., Katharina Hohenfellner, M.D., Maya Doyle

What is important to know about your health status and preparation for transition?

Review both chronic kidney disease and transplant medical conditions.

Identifying a medical home leader and appropriate partnering specialists.

Timing of transition and unique factors to consider, I.e., age, geographic location, special needs, etc

What specific medical/surgical themes need to be addressed?

Family planning and opportunities for assistance.


Lasalle Ballroom

3:15 pm -4:05 pm

Adult Issues/Management of Extra Renal Manifestations

Moderator: William A. Gahl, M.D., Ph.D. Panelists: Galina Nesterova, M.D., Rachel Bishop, M.D., Doris Trauner, M.D., Robert Kleta, M.D., Ph.D.


Lasalle Ballroom

4:10 pm -5:10 pm


Medical Panel

Moderator: Jess Thoene, M.D.

Please join the entire group for the unique and informative opportunity to have your questions and concerns addressed by the leading physicians and researchers in cystinosis. All of the doctors who have presented at the Family Conference, all attending Medical Advisory Board and Scientific Review Board members, as well as other health care professionals involved in treating and researching cystinosis are scheduled to participate. Questions for the panel will be collected during the proceedings today.




Lasalle Ballroom

5:10 pm –

5:40 pm


Above and Beyond Achievement Award / Group Photograph

Christy Greeley, CRN V.P. of Research and Executive Director

Lasalle Ballroom

7:00 pm


Speaker Dinner

Closed Session

Off Site

Saturday, July 18




Room


7:00 am –

8:30 am



Breakfast

Complimentary

Lasalle Ballroom

8:00 am –

5:30 pm


Registration/Information Desk

Lasalle Ballroom Lobby

8:00 am –

5:30 pm


Child Care Open

State

8:30 am – 9:00 pm

Teen Lounge Open


Room?




Dream, Achieve, Inspire” art exhibit

"Dream, Achieve, Inspire" an Art Exhibit Artists with cystinosis from around the world have come together to share their creativity for global cystinosis awareness. More than 80 artists from 29 countries have united to bring cystinosis struggles to light. Each piece tells a story of strength and hope. The exhibit crosses language and cultural barriers in an effort to express the need for research, better treatments and in some countries access to life saving medication where there is none. "Dream, Achieve, Inspire" is an emotional journey through the hearts of those with cystinosis...Enjoy the beautiful ride!



Lasalle Ballroom




Artists in Residence

Snow City Arts invites you to participate in the creation of a mural representing our conference. All attendees are encouraged to add their own twist to the project that will become part of the Art Exhibit and travel the world to other cystinosis conferences. Be a part of our artistic love letter to the cystinosis community! The final piece will be unveiled at the closing party.




Huron




Global Genes Teen Adventure

Michigan

8:30 am –

8:40 am



Opening Comments

Jeff Larimore, CRN President

Lasalle Ballroom

8:45 am –

9:15 am



KDIGO Cystinosis Conference Update

Craig B. Langman, M.D.

The process for a controversies conference sponsored by KDIGO will be discussed, and the one that occurred in Lisbon in December 2014 will be reviewed for topic content and methodology moving forward. An opportunity for input by the community will be reviewed. Specific highlights will be noted.




Lasalle Ballroom

9:20 am –

9:40 am


Raptor Pharmaceuiticals/Accredo Procysbi Update

Eric Mosbrooker, VP, Commercial Operations, Americas

Raptor Pharmaceuticals and Bill Martin, Vice President, Accredo

Eric and Bill will provide an update on the services available to Procysbi patients to make accessing the drug from the pharmacy easier and more streamlined.  Eric will discuss Raptor’s Patient Support team and Bill will update our community on steps Accredo has taken to improve and expand the services available to patients.  Both organizations are committed to continuously improving, and each would like to hear from us about what they can do for the cystinosis community.




Lasalle Ballroom

9:45 am –10:05 am

Sigma Tau Pharmaceuiticals/Walgreens Cystaran Update

Lesli King, Senior Product Manager, Valerie Paterno, Senior Manager Distribution Services Sigma Tau Pharmaceuticals, and Amber Walker, Director, Specialty Pharmacy Development Walgreen’s Specialty Pharmacy

Lesli, Valerie and Amber will provide an update on the services available for obtaining access to Cystaran via Walgreens Specialty Pharmacy. Common questions and questions from the community about Cystaran will be addressed. Sigma-Tau and Walgreens are committed to the continued support of the Cystinosis community. Working together we continue to improve and streamline the processes, ensuring the patients receive the most efficient and robust services.




Lasalle Ballroom

10:05 am– 10:20 am

Break

Lasalle Ballroom

10:25am –

11:10 am




Parents of Children and Adults with Cystinosis Panel

Moderator: Jennifer Caughlin. Panelists: Jen Wyman, Brett and Brittney LeBeau, Kevin and Jackie Keizer, Jill Morrill, Don and Doretta Hoffmann, Vic and Ina Gardener, Clinton and Annie Moore

Panel presentation during which parents of children and adults with cystinosis will answer prepared questions and address topics related to the use of coping mechanisms through the ups and downs that cystinosis brings related to not only developmental and transitional issues of daily life but also medical issues. Parents of individuals at every stage of the disease will be featured and will share how they have managed the variety of challenges they have faced. Audience participation will be encouraged.




Lasalle Ballroom

11:15 am -

12:00 pm


Living with Cystinosis Panel

Moderator: Maya Doyle. Panelists: Stephanie Sorenson, Paula Shal, Jennifer Logisci, Steve Schleuder, Katie Morrison, Christian Morales, Andrew Hoffmann, Shea Hammond

Panel presentation during which individuals living with cystinosis will answer prepared questions and address topics related to the use of coping mechanisms through the ups and downs that cystinosis brings related to not only developmental and transitional issues of daily life but also medical issues. Adults living with cystinosis will be featured and will share how they have recognized and lived to their full potential given the challenges they have faced. Audience participation will be encouraged.




Lasalle Ballroom

12:00 pm– 1:00 pm


Luncheon

Complimentary

Lasalle Ballroom

1:05 pm –

1:35 pm



Bullying and Youth with Special Healthcare Needs

Wilfred J. Krenn, Psychotherapist

Lasalle Ballroom

1:40 pm –

2:00 pm



Preimplantation Genetics/Early Pregnancy Diagnosis of Cystinosis

Jared C. Robins, MD, Associate Professor, Chief, Division of Reproductive Endocrinology and Infertility, Department of Obstetrics and Gynecology, Northwestern University Feinberg School of Medicine


Lasalle Ballroom

2:05 pm –

2:25 pm



Adherence Issues

Ewa Elenberg, M.D.

Why people are not compliant, spectrum of the problem, Relationship between compliance score and time to ESRD, Renal Fanconi Syndrome index




Lasalle Ballroom

2:30 pm –

2:50 pm


Rickets, Osteomalacia and Bone Deformities in Patients with Fanconi Syndrome of Cystinosis: Recent Data, Diagnosis and Therapies

Galina Nesterova, M.D.

Children with nephropathic cystinosis invariably develop renal Fanconi syndrome with predominately proximal tubular dysfunction, leading to the loss of electrolytes and minerals, particularly calcium and phosphate. If inadequately treated with supplementations of calcium, phosphate and vitamin D, this leads inexorably to rickets/osteomalacia, with dramatic skeletal deformities during growth periods due to insufficient accretion of hydroxyapatite. We have become aware that small but chronic losses of bone mineral, not recognized by laboratory studies such as serum phosphate, calcium, and alkaline phosphatase, can produce devastating bone disease.  One goal is to investigate, evaluate, and prevent bone loss and deformity due to hypercalciuria and hyperphosphaturia, and to develop appropriate therapeutic guidelines for preventing such consequences of Fanconi syndrome.




Lasalle Ballroom

2:50 pm –

3:10 pm



Break

Lasalle Ballroom Lobby

3:15 pm –

4:00 pm



Workshop Session A

These topic specific workshops allow families to tailor their conference experience to meet their specific needs. Families will have the opportunity to discuss and share their own experiences regarding the latest research, management techniques, and therapies with leading experts as well as with other families with similar issues and concerns. Sessions will be repeated so that participants may attend more than one workshop.




  1. Adult Session I (closed)

William A. Gahl, M.D., Ph.D., Galina Nesterova, M.D., Rachel Bishop, M.D., Doris Trauner, M.D., Robert Kleta, M.D., Ph.D.

General Issues




  1. Teen/Transition Session

Paul Grimm, M.D., Minnie Sarwal, M.D., Ph.D., Frederick Kaskel, M.D., Ph.D., Richard Simon, M.D., Patrick Gipson, M.D., Karen Waggenbrenner, Katharina Hohenfellner, M.D., Maria Helena Vaisbich, M.D., Elena Levtchenko, M.D.


  1. Newly Diagnosed Families/Childhood Session

Craig Langman, M.D., Paul Goodyer, M.D., Ewa Elenberg, M.D., Larry Greenbaum, M.D., Ph.D., Jess Thoene, M.D.



Superior I
Superior II
Superior III

4:15 pm –

5:00 pm



Workshop Session B


  1. Adult Session II (closed)

Moderator: Jennifer Coughlin. Panelists: William A. Gahl, M.D., Ph.D., Galina Nesterova, M.D., Jennifer Logisci

sex/reproduction/birth control issues



  1. Teen/Transition Session

Paul Grimm, M.D., Minnie Sarwal, M.D., Ph.D., Frederick Kaskel, M.D., Ph.D., Richard Simon, M.D., Patrick Gipson, M.D., Karen Waggenbrenner, Katharina Hohenfellner, M.D., Maria Helena Vaisbich, M.D., Elena Levtchenko, M.D.



  1. Newly Diagnosed Families/Childhood Session

Craig Langman, M.D., Paul Goodyer, M.D., Ewa Elenberg, M.D., Larry Greenbaum, M.D., Ph.D., Rachel Bishop, M.D., Jess Thoene, M.D.,

Doris Trauner, M.D.





Superior I

Superior II

Superior III

5:10 pm –

5:30 pm


Conference Wrap Up

Jeff Larimore, CRN President

Lasalle Ballroom


6:30 pm –

10:30 pm



Farewell Dinner Dance

Complimentary, Cash Bar. Please join all conference attendees for dinner, dancing, photo booth and fun for all ages to celebrate our cystinosis community. Special performance by Tori Lynn Jones.





Lasalle Ballroom


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