PPMD’s End Duchenne Tour made a stop in Memphis, TN on Saturday, October 26. We were joined by over 60 people,  including families, an amazing group of volunteers in our kids room, six members of the Le Bonheur Children’s Hospital team, and three members of the Children’s of Arkansas Team.

This group was engaged and interactive, lending to great discussion about the current landscape of Duchenne care and research.

Join the Fight

Race to End Duchenne

Amber Sapp, parent and runner, shared with the group about her experience participating in the Race to End Duchenne and how it’s helped her to get involved in the Duchenne community. She encouraged people to join the Race to End Duchenne team at the Great American River Run in Memphis on May 23, 2020, as part of the Memphis in May Festival!

PPMD’s Connect

Tammy Cate and Amber Sapp, Tennessee’s PPMD Connect coordinators discussed this parent-led outreach program that connects families within their region. Amber and Tammy talked about the importance of connecting with other parents, and shared information about their upcoming fall Connect get-together on Saturday, November 16 in Columbia, TN.

Presentation Highlights

Navigating the Challenges of School

Denise Gruender (ABC Educational Services)

• Denise met with individual families throughout the day to discuss learning and behavioral concerns.
• To reach Denise, you may email her office manager at erika.g@abcedservices.com or by phone: 704-443-2990.

Physical Therapy

Melissa Hicks-Wittman, PT, DPT (Arkansas Children’s Hospital)

• Melissa reviewed the role of a PT in Duchenne along with typical long term goals with which the PT can assist
• Focus was placed on the importance of stretching and positioning in maintaining flexibility and range of motion as long as possible
• Download presentation slides

About PPMD

Ryan Fischer (PPMD)

• Ryan spoke about the core pillars of PPMD, including the importance of advocacy, engagement, research, and care.
• Ryan shared information about the 2020 Advocacy Conference in Washington, D.C.
• Download presentation slides

Duchenne Research Overview: The Landscape and Opportunities

Dr. Aravindhan Veerapandiyan (Arkansas Children’s Hospital)

• Dr. Veerapandiyan introduced clinical trials, including the different pathways by which to access clinical trials and the different categories of clinical trials
• Dr. Veerapandiyan also discussed several of the different compounds currently being studied within the Duchenne pipeline
• Download presentation slides

Genetics, Your Family, and The Duchenne Registry

Ann Martin, CGC (PPMD)

• Ann provided a review of genetics and what different types of mutations do to the dystrophin gene.
• Ann also reviewed important changes to The Duchenne Registry and its new format.
• Download presentation slides

Neuromuscular Standards of Care

Rachel Schrader MS, APRN, CPNP (PPMD)

• Rachel reviewed how Duchenne effects not only the muscles, but the entire body
• Rachel discussed current care standards, including emergency care recommendations
• Download presentation slides

Duchenne Specialist Panel

• Jonathan Finder, MD (Le Bonheur Children’s Hospital) – Pulmonology
• Elena Caron, MD (Le Bonheur Children’s Hospital) – Neurology
• Hugo Martinez, MD (Le Bonheur Children’s Hospital) – Cardiology
• Aravindhan Veerpandiyan, MD (Arkansas Children’s Hospital) – Neurology
• Seth Sorensen, PhD (Arkansas Children’s Hospital) – Neuropsychology

Industry Updates

Catabasis

Elizabeth Higgins

• Edasalonexent currently in Phase 3 and open label extension studies
• Expecting results in Q4 of 2020 for potential NDA filing in early 2021
• Download presentation slides

Italfarmaco

Ana Christensen

• Phase 2 of givinostat showed delay of disease progression compared to natural history
• Phase 3 open now at sites around the world
• Download presentation slides

NS Pharma

Ana Christensen

• Phase 3 study of viltolarsen opening soon
• Download presentation slides

PTC

Donna Rollins

• Donna discussed the various programming PTC offers, including PTC Cares, which supports families and provides case management

Santhera

Jessa Depew

• Jessa reviewed the interaction of respiratory health and mitochondrial function
• Patients can access idebenone via Phase 3 SIDEROS trial or through expanded access program, BreatheDMD
• Download presentation slides

Pfizer

Helene Schluep

• Helene provided an update of Phizer’s microdystrophin candidate
• Phase 3 program expected to begin in 2020

Sarpeta

Kimber Hijek

• Sarepta is working to advance therapies including RNA, gene therapy, and gene editing with a total of 15 current programs for Duchenne muscular dystrophy
• Route 79 scholarship is available for those living with Duchenne who are attending college

Solid

Pat Gonzalez, PhD

• Corrective, disease modifying, understanding, assistive devices
• Their version of microdystrophin, SGT-001, is inclusive of all mutations
• Enrollment for IgniteDMD is ongoing, including new sites at UMass and Lurie.
• Download presentation slides

Wave

Allison Santoro

• Allison reviewed the concept of stereopure molecules
• DYSTANCE 51 is currently in phase 2/3 clinical trials.

Thank You!

Many thanks to the team at Le Bonheur Children’s Hospital, especially Dr. Johnathan Finder and his spectacular team, including Dr. Caron, Clinic Director, and Carrie Knappier, nurse coordinator, for hosting this event!

We also would like to thank Stacie Al-Chokhachi for recruiting volunteers who made the kid’s room so much fun!

See you in 2020!

This year we travelled to 5 cities across the US and launched our Spanish Remote Tour. We brought hundreds of families, providers, advocates, subject experts, industry partners and volunteers together to continue to educate and empower our community.

We thank each and every one of you that participated in our Tour this year. We’ll see you back on the road next year, with some exciting updates and enhancements!

 

Upcoming 2020 Schedule*

• Houston, TX Connect Session: January 18^th, 2020
• Atlanta, GA: March 14^th, 2020
• Milwaukee, WI: April 18^th, 2020
• Louis, MO: May 9^th, 2020
• Portland, OR: Fall 2020
• Miami, FL Connect Session: Fall 2020
• Long Island, NY: Fall 2020

*Registration typically opens 1-2 months prior to each event. Visit EndDuchenne.org/Tour for more details and make sure you are signed up to receive emails from PPMD to be notified when registration opens.