2024

AMDA/IPA

International
Pompe Patient and Scientific Conference

San Antonio, Texas
May 3rd – 5th, 2024

Agenda

SAVE THE DATE

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Registration is now open!

We are excited to announce that the 2024 AMDA/IPA International Pompe Patient and Scientific Conference will take place in San Antonio, Texas from May 3rd-5th, 2024.

There is no Conference Registration Fee for patients and their families. For Industry Representatives, please email info@amda-pompe.org for more information.

Register Virtual
Register In-person

AGENDA

This is a tentative agenda.  We are still in the process of finalizing sessions and speakers. As soon as more information is available, it will be posted in this website.

1:00-4:30 Workshops 

  • 1:00-2:00 First Session 
  • Mental Health
    • Caregivers Breakout Room—led by Julie Wells (Give an Hour)
    • Patient Breakout Room—led by Dr. van den Hout
    • Parent’s Breakout Room—led by Julienne Williams
  • 2:00-2:15 Break
  • 2:15-3:15 Second Session (Two Workshops)
    • How to monitor LOPD patients diagnosed through NBS—Dr. Cocanougher and Amanda Joost
    • Transitioning from Pediatric Care to Adult Care—Dr. van den Hout and Zach DuMond
  • 3:15-3:30 Break
  • 3:30 -4:30 Third Session (Two Workshops) 
    1) Exercise Recommendations for IOPD—Dr. Laura Case
    2) Exercise Recommendations LOPD—Dr. Linda den Berg

End of Workshops

  • 5:30-9:00 Welcome Dinner (meet in the Lobby of the Holiday Inn for departure)

7:00–8:00 Breakfast

  • 8:00-8:10 Welcome Address 
    8:10-8:40 Understanding Pompe: What Do We Know, and What Is Left To Learn by Drs. Arnold Reuser and
    Nina Raben
  • Natural History of Pompe vs. New History with ERT
    8:40-8:50 Chair: Dr. Priya Kishnani
    8:50-9:10 Infantile-Onset by Dr. Hannerieke van den Hout
    9:10-9:30 Late-Onset by Dr. Mark Roberts
    9:30-9:50 Effects of Newborn Screening by Dr. Nancy Chien
    9:50-10:05 Question and Answer
  • 10:05-10:25 Break
  • The Whole Patient—Monitoring of Patients
    10:25-10:30 Chair: Dr. Benedikt Schoser
    10:30-11:00 Integrative Care of Pompe Patients by Dr. Andreas Hahn
    11:00-11:15 Monitoring of Patients: Biomarkers by Dr. Giancarlo Parenti
    11:15-11:30 Monitoring of Patients: Muscle MRI by Dr. Jordi Diaz-Manera
    11:30-11:45 Monitoring of Patients: Muscle Ultrasound by Dr. Neha Regmi
    11:45-12:00 Question and Answer
  • 12:00 -1:00 Lunch 
  • Management and Care of Pompe Patients
    1:00-1:05 Chair. Dr. Ans van der Ploeg
    1:05-1:20 Diet approach by Dr. Dominic D’Agostino
    1:20-1:35 Exercise and Nutritional Considerations for the Treatment of Pompe Disease by Dr. Mark Tarnopolsky
    1:35-1:50 Non-Invasive Ventilation for Pompe by Dr. Grazia Crescimanno
    1:50-2:05 Respiratory Muscle training Project by Elena Compalati and Rosario Di Marco
    2:05-2:20 Speech Pathology by Dr. Harrison Jones
    2:20-2:35 Pain Management for Pompe by Dr. Heidi Peters
    2:35-2:50 Question and Answer
  • 2:50-3:10 Break 
  • Expert Roundtable: 25 Years of Treatment and Care of Pompe Patients
    Chair: Tiffany House
    (Dr. Priya Kishnani, Dr. Benedikt Schoser, Dr. Ans van der Ploeg, Dr. Mark Roberts)
    3:10-4:15 Open Discussion with Audience
  • Challenges of Managing Pompe-The Patient Perspective Roundtable
    4:15-5:15 Chair: TBD
    Panel TBD

End of Day 1

  • 6:30-9:00 Patient Reception 
  • 7:00-8:00 Breakfast
  • IPA/Erasmus Survey and Pompe Registries
    8:00-8:05 Chair: Brad Crittenden
    8:05-8:20 Report on IPA/Erasmus Pompe Survey/Mutation Database by Dr. Nadine van der Beek
    8:20-8:30 Report from Genzyme’s Pompe Registry by Dr. Tiziana Mongini
    8:30-8:40 Report from French Registry by Dr. Pascal Laforet
    8:40-8:50 Where Do We Go From Here and Why is it Important? By Dr. Benedikt Schoser
    8:50-9:05 Question and Answer
  • 9:05-9:25 Break 
  • 9:25-11:00 Next Generation Therapies: ERT, Gene Therapy and Substrate Reduction (All Industry) 
    9:25-9:35 Chair: Dr. Federico Mingozzi – ERT
    9:35-9:45 Amicus
    9:45-9:55 M6P 
    9:55-10:05  Sanofi Gene Therapy
    10:05-10:15 Introduction to Gene Therapy
    10:15-10:25 Astellas
    10:25-10:35 LentiCure
    10:35-10:45 Regeneron 
    10:45-11:00 Question and Answer Reduction Therapy
  • 11:00-11:20 Break
  • Future Research
    11:20-11:30 Chair: Dr. Ans van der Ploeg
    11:30–11:50 How to Fast Track Preclinical Testing New Treatments for Pompe disease by Dr. Nina Raben
    11:50-12:10 Substrate Reduction Therapy in Pompe by Dr. Virginia Kimonis
    12:10-12:30 PS Gene-editing for Pompe disease by Dr. Chester Whitley
    12:30-12:50 Stem Cell and Muscle Regeneration in Pompe by Dr. Pim Pijnappel
    12:50-1:00 Question and Answer

CONFERENCE CLOSE 1:00-1:30

Confirmed Speakers

We are excited to announce some of our confirmed speakers for the conference!

Dr. Laura Case – United States

Dr. Yin-Hsiu (Nancy) Chien – Taiwan

Dr. Benjamin Cocanougher – United States

Elena Compalati – Italy

Dr. Dominic D’Agostino – United States

Rosario Di Marco – Italy

Zack DuMond – United States

Dr. Andreas Hahn – Germany

Erin Huggins – United States

Dr. Harrison Jones – United States

Amanda Joost – United States

Dr. Priya Kishnani – United States

Dr. Pascal Laforet – France

Dr. Federico Mingozzi – United States

Dr. Tiziana Mongini – Italy

Dr. Giancarlo Parenti – Italy

Dr. Pim Pijnappel – the Netherlands

Dr. Nina Raben – United States

Dr. Arnold Reuser – the Netherlands

Dr. Mark Roberts – United Kingdom

Dr. Benedikt Schoser – Germany

Dr. Mark Tarnopolsky – Canda

Dr. Antonio Toscano – Italy

Dr. Linda van den Berg – the Netherlands

Dr. Hannerieke van den Hout – the Netherlands

Dr. Nadine van der Beek – the Netherlands

Dr. Ans van der Ploeg – the Netherlands

LOCATION

The conference will be held at the Holiday Inn Riverwalk from
May 3 – May 5, 2024.

217 North St Mary’s St
San Antonio, TX 78205-2303
(210) 224-2500

Note on Hotel Reservations with the host hotel:

If rooms at the host hotel run out, there are several hotels nearby. For a list of nearby hotels, click here.

To modify any existing group reservation at the host hostel, please work directly through Bonnie Ng.  You can either call or email Bonnie Ng directly.

*The AMDA will only pay for up to 3 nights at the host hotel for people registered with the AMDA*

Frequently Asked Questions

If you have any questions, we have put together a Frequently Asked Questions (FAQ). If you still have questions after reading the FAQs, please send us an email at: info@amda-pompe.org.

Is there a fee to attend the Conference?

No. There is no fee to attend the Conference for patients and their families / caregivers. Other attendees will be considered on a case-by-case basis.

Please send inquiries to info@amdapompe.org

I heard that the AMDA will pay for one hotel room per patient registered with the AMDA. How will this work?

The AMDA will pay for one hotel room per patient registered with the AMDA (at the rate of $229/night) for up to three nights. The third night is to support your participation in the Friday Workshops.

This is non-transferable. If you only need a room for 1 or 2 nights, you CANNOT give the other night(s) to someone else.
Also, there are limited funds available, so the funds will be made available based on the order in which people register for the Conference and only at the group rate of $229/night.

Finally, the AMDA will pay for the room charge ONLY– any room service, amenities, or other items charged to the room are the sole responsibility of the patient.

If you are not sure if you are registered with the AMDA, please email the AMDA Patient Advocate, Marsha Zimmerman, at
Marsha.Zimmerman@amda-pompe.org

Do I have to make my own hotel reservation?

YES, each patient is responsible for making his or her own
reservation with the hotel.

The conference will be held at the Holiday Inn Riverwalk from
May 3, 2024 – May 5, 2024.

If you plan to participate in the Friday (May 3rd) workshops, the AMDA will pay for one additional night at the hotel – May 2,
2024.

Here is the 2024 AMDA – IPA Conference booking link: CLICK HERE

Once you click on the link it will take you to the hotel website • Enter your arrival/ departure dates. • It will automatically populate your group name and discounted group rate.
You may also call the 24-hour reservation phone number at 210-224-2500, option 2. • Remember to reference the group’s block code: AMD

The last day to make reservations at the group discount will be: April 2, 2024.

To modify any existing group reservation, please work directly
through Bonnie Ng. You can either call or email Bonnie Ng directly:

How do I know if my hotel room will be paid for by the AMDA?

Funds will be made available based on the order in which people register for the Conference (please note that funds are only available to people who have registered with the AMDA).

When you receive your registration confirmation you will be told whether you have registered in time

What is the address for the hotel?

 Holiday Inn San Antonio – Riverwalk
217 North St Mary’s St
San Antonio, TX 78205-2303
(210) 224-2500

Will the AMDA be providing transportation to and from the airport?

No. Each guest is responsible for arranging his/her own transportation from the airport.
The San Antonio International Airport is located approximately 10 minutes from the hotel.

To assist you in making your arrangements, please consult the information below.

If you need a wheelchair-accessible taxi, it is advised that you
make your reservations early!

 

Can I bring my children to the conference? Will there be childcare?

Children are welcome to come to the conference if you would like them to be there.
There will be a “family room” that will be located close to the conference room.
No babysitting will be provided, but there will be someone available to help with crafts, and other activities for children to
do.
Please know that in the past, some children have not been too keen to stay in the family room and wanted to be with their
parents.

 

Where should I go to get to the welcome dinner Friday night?

The Welcome dinner will be held at a special offsite location with activities, photo ops, and entertainment:

  • Transportation to and from the event will be provided.
  • Everyone will meet in the lobby no later than 5:30 pm 
  • The buses will leave the event to return to the hotel at 9pm.
  • Casual Western wear is recommended

Note: If attending the Exercise Recommendations for IOPD or LOPD Workshops on May 3rd/Friday afternoon, it is recommended to bring/wear fitness/workout attire.

 

Will there be something for the conference participants to do on Saturday night?

Yes, the AMDA will have a reception (6:30 pm — 9 pm) in the Atrium where the conference participants can network with industry and get to know each other better:

  • Karaoke
  • Heavy hors d’oeuvres
  • Cash bar

Further details will be provided at the conference.

 

Ready to Register ?

BECOME A SPONSOR

Please see the Sponsorship Opportunities PDF for Sponsorship opportunities that are available.

To discuss participating as a Sponsor of the Conference, please contact: info@amda-pompe.org.