Rare Disease Day 2019

rdd2019

Clinical Gene Therapy: The Future is Now

Thank you for all who joined us!

This year's program held on March 1, 2019, furthered our understanding of gene therapy by hearing from a clinician and a panel of patients and parents. The clinician provided an overview of current therapies and therapies that are in the pipeline, whereas the panel featured gene therapy patients and parents of children who are potential recipients.  All were welcomed to this free event.

Watch the Presentations Now

Rare Disease Day 2019 - Clinical Gene Therapy: The Future is Now

Rare Disease Day 2019

Patient panelists

Patient panelists Pam Mertz, Mohamed Duklef, Ashanthi De Silva, Dr. Wilson, and Jack Keimel.

Round table discussions at the Patient Advocacy Group Breakfast.

Round table discussions at the Patient Advocacy Group Breakfast.

Round table discussions at the Patient Advocacy Group Breakfast.

Round table discussions at the Patient Advocacy Group Breakfast.

Round table discussions at the Patient Advocacy Group Breakfast.

Round table discussions at the Patient Advocacy Group Breakfast.

Round table discussions at the Patient Advocacy Group Breakfast.

Round table discussions at the Patient Advocacy Group Breakfast.

2019 Rare Disease Day Research Award Winners

2019 Rare Disease Day Research Award Winners and College of Pharmacy Dean Lynda Welage. (l-r Sarah Kim, Irene Vuu, Afroz Mohamma

College of Pharmacy Dean Lynda Welage

College of Pharmacy Dean Lynda Welage announces the 2019 Rare Disease Day Research Award winners.

Dr. Wilson gives his keynote address.

Dr. Wilson gives his keynote address.

Attendee listens to the keynote address by Dr. Wilson.

Attendee listens to the keynote address by Dr. Wilson.

Erica Barnes introduces the patient panel.

Erica Barnes introduces the patient panel.

Patient panelists listen as panelist Mohamed Duklef speaks.

Patient panelists listen as panelist Mohamed Duklef speaks.

Patient panel moderator, Patti Engel.

Patient panel moderator, Patti Engel.

Patient panelist, Ashanthi De Silva.

Patient panelist, Ashanthi De Silva.

Round table discussions at the Patient Advocacy Group Breakfast.

Round table discussions at the Patient Advocacy Group Breakfast.

Poster presenters hanging up their research poster.

Poster presenters hanging up their research poster.

Attendees interacting with poster presenters.

Attendees interacting with poster presenters.

Attendees interacting with a representative from the Rare Action Network.

Attendees interacting with a representative from the Rare Action Network.

Attendees interacting with representatives from Engage Health, Inc. and the Pompe Warriors Foundation.

Attendees interacting with representatives from Engage Health, Inc. and the Pompe Warriors Foundation.

Poster presenters hanging up their research poster.

Poster presenters hanging up their research poster.

Attendees interacting with representatives from the Epilepsy Foundation of Minnesota.

Attendees interacting with representatives from the Epilepsy Foundation of Minnesota.

Attendees interacting with poster presenters.

Attendees interacting with poster presenters.

Attendees interacting with poster presenters.

Attendees interacting with poster presenters.

Attendee interacting with a representative from the National Ataxia Foundation.

Attendee interacting with a representative from the National Ataxia Foundation.

Attendees interacting with a representative from the Paul and Sheila Wellstone Muscular Dystrophy Center.

Attendees interacting with a representative from the Paul and Sheila Wellstone Muscular Dystrophy Center.

Attendees interacting with representatives from the Organic Acidemia Association and the United Mitochondrial Disease Foundation

Attendees interacting with representatives from the Organic Acidemia Association and the United Mitochondrial Disease Foundation

Attendee interacting with a representative from the Rein in Sarcoma Foundation.

Attendee interacting with a representative from the Rein in Sarcoma Foundation.

Attendees interaction with a representative from the National Ataxia Foundation.

Attendees interaction with a representative from the National Ataxia Foundation.

Representatives from the United Mitochondrial Disease Foundation.

Representatives from the United Mitochondrial Disease Foundation.

Poster presenter, Todd Vanyo.

Poster presenter, Todd Vanyo.

Representative from the National Ataxia Foundation.

Representative from the National Ataxia Foundation.

Representatives from the Prader-Willi Syndrome Association.

Representatives from the Prader-Willi Syndrome Association.

Attendees interacting with event sponsors.

Attendees interacting with event sponsors.

Medical School Dean Jakub Tolar making opening remarks and introducing Dr. Wilson.

Medical School Dean Jakub Tolar making opening remarks and introducing Dr. Wilson.

Dr. Wilson giving his keynote address.

Dr. Wilson giving his keynote address.

Attendees listing to Dr. Wilson's keynote.

Attendees listing to Dr. Wilson's keynote.

rdd logo

Senator Amy Klobuchar

Senator Amy Klobuchar Rare Disease Day 2019

Senator Tina Smith

Senator Tina Smith Rare Disease Day 2019

Co-Sponsors of Rare Disease Day

Center for Orphan Drug Research
University of Minnesota Medical School Stem Cell Institute

Keynote

keynoteJames M. Wilson, MD, PhD

Rose H. Weiss Professor and Director
Orphan Disease Center, University of Pennsylvania

Panel

Patient Perspectives on Gene Therapy - Patients and caregivers representing several rare disease communities share their thoughts, hopes and concerns regarding gene therapy.