Calendar of Events

The listing of any event, or information about any event, or external web page(s) on the Lysosomal Disease Network web site does not imply or express any endorsement or guarantee of that event, that event information, or of any information or hyperlink on that external web page.

American Society of Gene and Cell Therapy
22nd Annual Meeting

April 29–May 2, 2019 in Washington, D.C., USA

ASGCT’s 22nd Annual Meeting will take place at the Washington Hilton. Early-bird registration rates are available through April 8, 2019, and groups of five or more are eligible for an additional 15 percent discount. Non-members are invited to apply for membership when they register, to earn the member discount on their registration. Abstract submission deadline was January 16, 2019. Importantly, there will be no Late Breaking Abstract submission process for the ASGCT 22nd Annual Meeting. Associate Members who are the first and presenting author on an abstract will receive free registration for the ASGCT 22nd Annual Meeting. Attendee registration and hotel room reservations can be made now. Make your hotel reservations as soon as possible, because the housing block will sell out well before the Annual Meeting. After the ASGCT block is completely full, there’s no guarantee attendees will be able to get the same room rate during the meeting.

Meritorious Abstract Travel Awards: ASGCT will be offering Meritorious Abstract Travel Awards to Associate Members (students or postdoctoral fellows) who have submitted abstracts and received an average review score of between 1 and 3.5. Submitters must be Associate Members at the time of submitting their abstract to qualify for Travel Awards. There is not a separate application for travel grants. Submitters must sign-up for a travel grant during the abstract submission process.

Poster Production Funding: In 2019, ASGCT will once again pay for poster production for 30 Associate Members. Submitters must be Associate Members at the time of submitting their abstract to qualify for poster production funding. Poster production recipients will be chosen based on abstract review scores and a determination of the ASGCT Abstract Review Committee. Poster production for those selected will be through an online system.

Underrepresented Minority Travel Awards: ASGCT will offer a limited number of Underrepresented Minority Travel Awards for students and postdoctoral fellows submitting abstracts for the meeting. The top candidates for this award, based on average score, will receive a $500 travel award to the 22nd Annual Meeting. Candidate must be a U.S. resident and self-identify as belonging to one of the following groups: African American, Pacific Islander, Hispanic, Native American, Inuit, or Aleut. Candidate must be an Associate Member of ASGCT and the first and presenting author of an abstract being submitted for the 22nd Annual Meeting. After reading their web pages, you may e-mail the ASGCT for more information.

RARE on the Road

May 4, 2019 in Birmingham, Alabama, USA

The EveryLife Foundation and Global Genes® co-host this rare disease leadership tour. This traveling leadership and educational series helps equip attendees with the knowledge of how to advocate for yourself; to advocate in your local and rare disease community; and to advocate on a legislative level. The events aim to activate the rare disease community at a local level, and empower patients and caregivers with knowledge and insight to better serve their rare community. In addition to May 4, 2019 in Birmingham, the event is touring to:

• May 18, Denver, Colorado
• July 13, Sioux Falls, South Dakota

The EveryLife Foundation and Global Genes® have been making some limited $150 travel stipends available for each event. The Stipend Application deadline for Sioux Falls, South Dakota is Friday, June 14, 2019.

6th Update on Fabry Disease:
Biomarkers, Progression and Treatment Opportunities

May 26-28, 2019 in Prague, Czech Republic

Kidneys for Life (of the Manchester Royal Infirmary in Manchester, UK) is the supporting organization of the 6th Update on Fabry Disease. Charles University and its First Faculty of Medicine are the hosts for this meeting, which will focus on emerging treatment approaches, baseline characteristics that determine the patient-specific responses to the treatment of Fabry disease, and antibody interactions with infused enzyme replacement therapy. The meeting’s program is available (scroll down). The official language of the meeting is English.

Late registration began April 1, 2019. Late registrants will be accepted on a “first-come, first-served” basis with regards to registering for the conference and securing accommodation at the conference site. The maximum number of registrants will be capped at 120. The late registration fee includes lunch and breaks at the conference, but does not include hotel accommodations. The late-registration fee is 450 Euros. The conference convenes at InterContinental Hotel in Prague. All registrants will be required to book their accommodations at this hotel. All hotel rooms are booked through the conference’s Registration Site (scroll down for online registration form). These conference-block hotel rooms include breakfast.

2019 Krabbe Translational Research Network Meeting

May 29-31, 2019 in Minneapolis, Minnesota, USA

The Legacy of Angels Foundation is co-hosting, with the Lysosomal Disease Network, the 2019 Krabbe Translational Research Network (KTRN) meeting, which will convene on the campus of the University of Minnesota in Minneapolis. Questions can be directed to Stacy Pike-Langenfeld, Director of Programs and Administration for The Legacy of Angels Foundation.

Rare Drug Development Symposium

June 6-7, 2019 in Philadelphia, Pennsylvania, USA

Global Genes® is partnering with the University of Pennsylvania Orphan Disease Center to host this fourth annual RARE Drug Development Symposium, which was developed to help attendees learn about the drug development process. It is being held in conjunction with UPenn’s 6th annual Million Dollar Bike Ride. Attendees do not need to be affiliated with the Million Dollar Bike Ride, and all rare patients, advocates and stakeholders are welcome to attend.

The theme of this year’s RARE Drug Development Symposium is “Don’t Stop Innovating: Patients’ Role in Breakthrough Ideas.” The poster abstract submission period opened March 1, 2019, and closed April 4, 2019. All poster presenters will receive a certificate of participation. Additionally, the top three posters will be invited to present at the Global Genes RARE Patient Advocacy Summit and will be provided with a free registration. Scroll-down on the Rare Drug Development Symposium web page for sponsorship and exhibitor information.

6th Annual Million Dollar Bike Ride

June 8, 2019 in Philadelphia, Pennsylvania, USA

The Penn Medicine Orphan Disease Center (ODC) will host the 6th Annual Million Dollar Bike Ride on Saturday, June 8th, 2019 to raise money for rare disease research. The bike ride begins at 7:30 a.m. Individual cyclists and disease teams can register. To join a team, click on the name of the team on the disease teams web page. For example, to join the National MPS Society’s ‘Team MPS,’ click on ‘Team MPS’ below the MPS Society’s logo on the disease teams web page, and follow the links to the ultimate page for ‘Team MPS’ at the National MPS Society’s web site. There, you can easily send an e-mail to Brian Buechler at the MPS Society, for information about joining Team MPS.

A limited number of bicycle rentals are available from two different businesses located relatively close to the Million Dollar Bike Ride location. These rentals are available on a “first-come, first-served” basis, so don’t delay, reserve your bicycle today!

Again this year, Million Dollar Bike Ride has partnered with “Homewood Suites by Hilton” to provide a special room-rate for Million Dollar Bike Ride participants (cut-off date: May 26, 2019). A block of rooms at “Homewood Suites by Hilton” has been reserved for June 6 – June 9, 2019. (The special room-rate will be available until May 26th, or until the group block is sold-out, whichever comes first.) Homewood Suites Hotel is located just a few blocks away from the starting line, and is close to 30th Street Station.

Special room-rates for Million Dollar Bike Ride participants are also available from The Philadelphia 201 Hotel (cut-off date: May 7, 2019) and Sheraton Philadelphia University City Hotel (cut-off date: May 6, 2019). Follow the links on the accommodations page to learn more, and to make your hotel reservation.

After absorbing the information on the Million Dollar Bike Ride web site, you may contact for answers to any additional questions that may arise.

2019 Parseghian Classic at
Pebble Beach

June 21-24, 2019 at California’s Monterey Peninsula, USA

The 2019 Parseghian Classic, a golf fundraiser benefiting Niemann-Pick disease type C research at University of Notre Dame, will be held at Pebble Beach Resorts, in California. There are four differently-priced event packages to choose from, and non-golfers are also welcome. Event Registration fees include lodging for Friday, Saturday and Sunday at The Lodge at Pebble Beach™ and all scheduled activities. Kim Kirkpatrick at Notre Dame is available to answer your questions or to pre-register you via e-mail, or by telephone at (574) 631-3136.

MPS Society of the UK
2019 Annual Conference

June 28-30, 2019

It is time to register for the MPS Society of the UK’s 2019 Annual Conference. This year’s Saturday session offers three separate conferences running in parallel and focusing on:

• CONFERENCE A — Focuses on pediatrics
• CONFERENCE B — Focuses on young people and adults
• CONFERENCE C — Focuses on those with CNS involvement.

Each conference will have a mix of professional and patient talks, as well as panel discussions after each session.

The conference venue is the Doubletree by Hilton Hotel Coventry. If the conference bedrooms at the Doubletree by Hilton Hotel are full, alternative accommodation has been booked with the Holiday Inn, which is located very close to the Doubletree by Hilton Hotel. Early booking is recommended.

Little People of America
62nd Annual National Conference

June 28–July 5, 2019 in San Francisco, California, USA

The 2019 Annual LPA National Conference will convene at the Marriott Marquis Hotel located at 780 Mission Street in San Francisco. 2019 LPA Conference registration and hotel reservations are now available. Attendees must register with LPA first, and obtain a Registration ID number, in order to book their hotel room at the San Francisco Marriott Marquis. As new information becomes available it will be found on the LPA web site.

2019 Batten Disease Support and Research Association
Annual Family Conference

July 18-21, 2019 in Denver, Colorado, USA

Save the date for the BDSRA’s 2019 Annual Family Conference, which will convene at the Renaissance Denver Stapleton Hotel in Denver, Colorado. Visit for ongoing conference updates.

2019 Hunter’s Hope Annual Family and Medical Symposium

July 24–28, 2019 in Holiday Valley Resort, Ellicottville, New York, USA

Hunter’s Hope, which engages in vigorous leukodystrophy patient advocacy and supports research for leukodystrophy treatment, states that the annual Family and Medical Symposium “is a place where everyone understands and life-long friendships can be formed with other families on a similar path. There is no judgment, only love. For families in attendance, meals and accommodations are paid for by Hunter’s Hope. 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.” The Hunter’s Hope annual Family and Medical Symposium brings together the world’s leading leukodystrophy experts with families affected by these diseases, for a week of learning, sharing and fun.

Registration for the 2019 Family and Medical Symposium is NOW OPEN and will close on May 15th, or when capacity is filled, whichever happens first. For information on how to register for the 2019 Family and Medical Symposium, please contact Hunter’s Hope by e-mail at

ISMRD — The International Advocate
for Glycoprotein Storage Diseases
6th Glycoproteinoses International Conference

July 25-27, 2019 in Atlanta, Georgia, USA

ISMRD’s 6th Glycoproteinoses International Conference is themed “Translating Scientific Discovery into Therapies.” The ISMRD states, “This conference will once again bring basic scientists, clinicians and families together to share knowledge and inspiration as we continue towards the goal of translating basic science discoveries into therapies for the glycoproteinoses. We strive to highlight the most current advances in research and to discuss openly how these advances can be developed in treatments that will improve the quality of life for patients and families.”

The conference will convene at the Atlanta Marriott Alpharetta. Early bird registration closes April 30, 2019. You can e-mail the ISMRD with questions not addressed by their conference web pages.

The ISMRD is an internationally focused not-for-proft organization whose mission is to advocate for families and patients affected by one of the following disorders: alpha-mannosidosis, beta-mannosidosis, aspartylglucosaminuria, fucosidosis, galactosialidosis, sialidosis (mucolipidosis I), mucolipidosis II, II/III, or III alpha/beta, mucolipidosis III gamma, and Schindler disease.

RARE Patient Advocacy Summit

September 18-20, 2019 in San Diego, California, USA

Global Genes® ‘RARE Patient Advocacy Summit‘ gives rare disease stakeholders the opportunity to connect and learn through educational sessions and networking opportunities. Attracting more than 800 attendees, it is the largest gathering of rare disease patients, advocates and thought leaders worldwide. This year’s summit convenes at Sheraton San Diego Hotel & Marina.

Online registration is available now. There are significant discounts for those who register prior to 5:00 p.m. on May 15, 2019. Global Genes® is now accepting online applications for travel awards. Exhibitors can book their booth space now. Contact Global Genes® for answers to your exhibiting questions.

2019 AGSD Patient/Family/
Medical Conference

September 20-21, 2019 in Houston, Texas, USA

The 2019 Association for Glycogen Storage Disease family/medical conference for those affected with GSD, their families, and medical professionals involved in treatment or research of any type of GSD, is in the planning stages at this time. The focus of this annual conference is meeting other people and families affected by GSD, gaining a better understanding of the GSDs and their implications, and learning about the latest research findings and upcoming studies. The AGSD conference costs include a registration fee and the meal costs for Friday dinner and Saturday lunch. The Saturday evening dinner and entertainment is provided free for all conference registrants. Their web page will be updated as new information becomes available.

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

The listing of any event, or information about any event, or external web page(s) on the Lysosomal Disease Network web site does not imply or express any endorsement or guarantee of that event, that event information, or of any information on that external web page.

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