Calendar of Events

The listing of any event or web page on the Lysosomal Disease Network web site does not imply or express any endorsement or guarantee of that event or of any information on that web page.

Technology & Rare Neurological Diseases Symposium

May 12, 2017, 7:30 a.m. – 3:45 p.m.

Location: University of Rochester in Rochester, New York
Saunders Research Building, Helen Wood Hall Auditorium

The University of Rochester’s Center for Human Experimental Therapeutics is hosting and sponsoring a one-day symposium on novel applications of technology to accelerate the development of new therapeutics for patients with rare neurological disorders. Regional and national leaders in rare diseases, health technologies, and clinical trials plan to demonstrate how to leverage new tools and technologies to enhance patient partnership, reduce the burden of participation in clinical research, and accelerate development of new therapeutics. Presenters include patient advocacy group representatives, academic researchers in rare diseases, and pharmaceutical industry professionals. Here are all the details, and a link to register for this meeting. Or send an e-mail to Mulin Xiong at the University of Rochester.

5th Fabry Expert Meeting

May 19–20, 2017, at the Royal Olympic Hotel, Athens, Greece

Organized and hosted by the Fabry International Network, the 5th Fabry Expert Meeting is an opportunity for experts to discuss clinical aspects of Fabry disease, as well as to learn about innovative treatments and personal family experiences. The meeting weekend includes Friday industry meeting, Friday and Saturday night accommodation, Friday dinner, Saturday lunch and dinner, and Saturday expert meeting.

The Fabry International Network’s mission is to be a global, independent network of Fabry disease patient organizations whose purpose is to collaborate, communicate and promote best practices in support of those affected by Fabry disease. More information about the 5th Fabry Expert Meeting is available by e-mailing Toni, or calling +44 (0) 0345 389 9901 in the United Kingdom.

4th Annual Million Dollar Bike Ride

An Event of The Penn Medicine Orphan Disease Center

Saturday, May 20, 2017, from 7:30 a.m.- 4:00 p.m.

Location: Highline Park, 3160 Chestnut Street (31st Street and Chestnut) in Philadelphia, Pennsylvania, USA
Numerous LDN patient advocacy groups have intrepid teams participating in this world-famous rare disease fundraising event, including: Team Cure ML4, Team NTSAD, Team MPS, and Team NPC. No administrative cut is taken; 100% of money raised goes directly into rare disease research. There are over 600 participants. Over the past three annual Million Dollar Bike Rides, 17,538 miles have been bicycled. Are you up for the challenge? Online registration for the 2017 Million Dollar Bike Ride is open until midnight on Sunday, May 14. Walk-up registration is available at the Class of 1923 Penn Ice Rink on Friday, May 19 from 4-7 p.m. (3130 Walnut Street, Philadelphia, PA 19104); and on Saturday, May 20 at 6:00 a.m. Walk-up registration fee is $100. Learn more and register, or learn about sponsoring teams at: 4th Annual Million Dollar Bike Ride. Questions? Call 215-573-6822, or e-mail Million Dollar Ride.

2017 Canadian Association of Pompe Conference

June 10, 2017, at the Sheraton Centre Toronto Hotel, Toronto, Ontario, Canada

Planned topics at the 2017 Canadian Association of Pompe Conference will be: a Pompe disease Q & A with Dr. Mark Roberts; Ethics and Genetic Privacy; Mental Health; and Nutrition. For details, use the ‘Contact’ link on the Conference page. Visit their Facebook page as well.

4th International
Glycogen Storage Disease Conference
(“IGSD 2017”)

June 15-17, 2017 in Groningen, Netherlands

IGSD 2017 welcomes health care professionals (scientists, physicians, dietitians, students), and also glycogen storage disease patients, family members and patient advocacy group representatives. The official language of the IGSD 2017 is English. A highly-detailed preliminary program is available. The early-bird registration ended on February 15, 2017. The deadline for IGSD Scholarship applications was March 1, 2017. All IGSD Scholarship applicants will be notified of the outcome of their application by April 15, 2017. The conference venue is University Medical Center Groningen.

Batten Disease Support and
Research Association
(BDSRA)
2017 Family Conference

July 6-9, 2017, in Pittsburgh, Pennsylvania, USA

The 2017 BDSRA Family Conference will be at The Wyndham Grand Pittsburgh Downtown hotel. This year is the BDSRA’s 30th anniversary, and they would love to see old photos and hear stories that past attendees may have. Please e-mail for more information about sharing the amazing past of the BDSRA! Visit their Facebook page for ongoing updates.

The MPS Society (UK)
MPS Weekend Conference 2017

July 7-9, 2017 in the Doubletree by Hilton Hotel Coventry, Coventry, UK

This unique conference is dedicated to sharing the most up-to-date information available on the clinical management and treatment of MPS diseases, Fabry disease and related diseases, as well as clinical trials and research. This conference is open to all those living with or affected by MPS diseases, Fabry disease or related diseases, as well as health care, social care and educational professionals. This is an unmissable UK event for those personally or professionally involved in caring for a child or adult with MPS, Fabry or a related disease.

The Saturday and Sunday programs offer three separate conferences running in parallel and focusing on:
• Conference A: MPS I; MPS II (not progressive CNS); MPS IVA; MPS VI; LAL D (late onset); ML III and Mannosidosis
• Conference B: MPS II (progressive CNS); MPS IIIA, MPS IIIB, MPS IIIC, MPS IIID; LAL D (infantile); and rare related diseases
• Conference C: Fabry disease

To make your official reservation and learn much more, download the conference brochure/reservation form: MPS Society UK – July Conference Brochure or telephone 0345 389 9901. In addition to the complete agendas for all three parallel conferences, this brochure also provides in-depth information about the MPS Society’s extensive childcare program at this weekend conference for all children under 18 years old, as they are not permitted into the conference. Vulnerable young adults are also welcome to participate in this very special social childcare program packed full of fun and stimulating activities and outings. The Society hopes this will allow parents and carergivers to attend the conference in a relaxed manner, knowing that their children are safely cared for and well entertained.

The conference registration deadline is Friday, May 19, 2017. Attendees will want to register well in advance of that, since the hotel’s room offerings are on a first-come, first-served basis.

The MPS Society’s Upcoming Events & Clinics web page provides a wealth of information about all of the Society’s upcoming events!

The Cystinosis Research Network
2017 Family Conference

July 13-15, 2017 at the Snowbird Ski and Summer Resort, Snowbird, Utah, USA

The Cystinosis Research Network invites you to join them and learn about the latest updates in cystinosis research through lectures, workshops, poster sessions, and medical and patient panels. There will also be opportunities to socialize with other patients and families which will provide valuable connections and friendships that can last a lifetime. Registration materials will be available in early 2017 on the Cystinosis Research Network web site and will also be mailed to member families.

Hunter’s Hope Foundation
20th Annual Family & Medical Symposium

July 17-23, 2017 at Holiday Valley Resort in Ellicottville, New York, USA

The Hunter’s Hope 20th Annual Family & Medical Symposium brings together the world’s leading leukodystrophy experts with families affected by these diseases for a week of learning, sharing and fun. For families in attendance, meals and accommodations are paid for by Hunter’s Hope Foundation. Travel arrangements to and from the event are the responsibility of the family, but travel assistance is available for qualified families unable to cover these expenses. For more information about this, or other questions regarding the Family & Medical Symposium, please contact their Family Programs Director, Kathleen Scott.

The National MPS Society (U.S.)
Annual Family Conference

August 3-5, 2017, in Minneapolis, Minnesota, USA

The National MPS Society of the USA will convene its annual Family Conference at the Hilton Downtown, 1001 Marquette Avenue South, Minneapolis, Minnesota, 55403. On Saturday August 5 the Society will host an early morning 5K Walk/Run on the beautiful University of Minnesota campus. Conference details are still being developed; stay tuned at the MPS Society’s web site, and save the dates!

13th International Congress of
Inborn Errors of Metabolism

September 5-8, 2017, in Rio de Janeiro, Brazil

The 13th International Congress of Inborn Errors of Metabolism is accepting abstract submissions until May 5, 2017. Registration is open! You may e-mail your questions to info@nulliciem2017.org for more information.

Global Genes
RARE Patient Advocacy Summit 2017

September 14-15, 2017, in Irvine, California, USA

The 6th annual RARE Patient Advocacy Summit will convene at Hotel Irvine, 17900 Jamboree Road, Irvine, California, 92614. Global Genes is accepting travel scholarship applications for attendance at the RARE Patient Advocacy Summit 2017 through July 1st, 2017. Travel scholarships can be up to $600 for patients or family advocates in the rare disease community, and/or staff (paid or volunteer) of rare disease non-profit organizations or support groups. Registration is open!

5th Glycoproteinoses
International Conference
Hosted and Organized by the ISMRD

November 1-4, 2017 in Rome, Italy

Dr. Alessandra d’Azzo is the primary investigator for this scientific conference to be held in Rome, Italy. Dr. d’Azzo wrote, “Like earlier ISMRD gatherings, the conference will bring together basic scientists and clinicians from around the world to share with patient families and colleagues their latest discoveries in the areas of glycoproteinoses pathophysiology, investigational and pre-clinical therapy development, and clinical trials for these rare disorders.” Explore more information: about the conference venue; to make the hotel reservation; or to register & pay the fees. Children and adults diagnosed with a glycoprotein storage disease are admitted free of charge. Early-bird registration ends August 30, 2017. An outstanding lineup of international physicians and researchers comprise the scientific committee. The organizing committee is also quite impressive. More information will be posted at the conference’s web site as the conference draws closer.

15th International Symposium on MPS
and Related Diseases

August 1-4, 2018 in San Diego, California, USA

This is being organized by the International MPS Network. The International MPS Network is the official body of the patient associations representing MPS and MPS societies at a European and global level. Membership in the International MPS Network is only open to the one nationally recognised MPS Society or relevant patient association for each country globally. Up to two representatives from each accepted MPS Society or relevant patient association may attend the International MPS Network Meetings. There is no membership fee; however, member organizations are responsible for meeting the costs of their representatives’ attendance. Save the dates! Meeting details are not yet released. Check the web site periodically.


If you would like your event listed here, please send it to the web master.

The listing of any event or web page on the Lysosomal Disease Network web site does not imply or express any endorsement or guarantee of that event or of any information on that web page.


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