End Duchenne Tour: Denver, CO

PPMD was thrilled to spend Saturday, November 4th, in Denver, CO, with over 50 families, clinicians, researchers and industry partners discussing Duchenne care, research and daily living. Conversations were not only focused on updates in research and care resources, but focused on the lived experiences of families and individuals affected by Duchenne and Becker. 

Families were able to share their perspectives on clinical trials, clinic visits, finding peer support, recreational therapy, adaptive sports, and daily quality of life. The afternoon session was led by Children’s Hospital of Colorado’s Dr. Lauren Treat, Palliative Care physician, Jesica Ray, Social Worker, Haley Bevan, from Adaptive Recreation for Childhood Health (ARCH), and Kennan Gluck, member of PPMD’s Adult Advisory Committee (PAAC). 

The day ended with a social gathering led by PPMD’s local Connect Coordinators where families had a chance to relax, share stories and connect with one another.

PPMD strives to facilitate community connection and support for individuals and their families through our in person events as well as virtual opportunities. We are excited to expand our efforts during our PPMD Together events in 2024. Together we are stronger.

If you are looking for support, the PPMD staff is available to meet with families individually and we welcome you to schedule a meeting through PPMD For You.

PPMD is always here to help families make connections with others, and PPMD’s local Connect Groups are a great place to start.

Pre-Recorded Presentations

In effort to prioritize discussion at our in-person tour stop, we have included full presentations to reference below. These may be helpful to reference:

Genetics

Questions?
coordinator@parentprojectmd.org

Avidity Biosciences

Learn more about Avidity’s Phase 1/2 EXPLORE44 clinical trial investigating AOC 1044, a potential exon skipping therapy for patients with Duchenne muscular dystrophy amenable to exon 44 skipping

Catalyst

Learn more about AGAMREE (Vamorolone), which was recently approved by the FDA for use in patients with Duchenne muscular dystrophy ages 2 years and older

Edgewise Therapeutics

Learn more about Edgewise’s Phase 2 GRAND CANYON study investigating EDG-5506, a small molecule muscle stabilizer, in patients with Becker muscular dystrophy

NS Pharma

Learn more about NS Pharma’s approved therapy, VILTEPSO, an antisense oglionucleotide for individuals with Duchenne muscular dystrophy amenable to exon 53 skipping and their patient support program, NS Support

Pfizer

Pfizer is currently running an ongoing Phase 3 clinical trial investigating PF-06939926, a gene therapy for patients with Duchenne muscular dystrophy

PTC Therapeutics

Learn more about PTC’s approved therapy, EMFLAZA, a corticosteroid, and their patient support program, PTC Cares

REGENXBIO

Learn about REGENXBIO ‘s Phase 1/2 clinical trial, AFFINITY DUCHENNE™, for their investigational gene therapy, RGX-202 for Duchenne muscular dystrophy and AFFINITY BEYOND™, an AAV8 antibody assessment study.

Sarepta Therapeutics

Learn more about Sarepta’s patient support program, SareptAssist, to access currently approved therapies (AMONDYS 45, EXONDYS 51, VYONDYS 53, and ELEVIDYS), and ongoing clinical trials investigating potential therapies for people with Duchenne muscular dystrophy amenable to exon 51 skipping

Clinical Trial Overview

Questions?
eric@parentprojectmd.org