End Duchenne Tour: Chicago, IL

On Saturday, October 7th, the PPMD team spent the day in Chicago with over 50 family members, clinicians, and sponsoring industry partners. The morning was spent discussing care resources, genetics, research participation, and school planning.

Thank you to Dr. Bridget McGowan from Lurie Children’s Hospital Chicago, a local PPMD Certified Duchenne Care Center, and Jill Castle, M.Ed, an education and behavioral specialist, for sharing their knowledge, experience, and guidance.

The PPMD staff is available to meet with families individually to discuss these topics, and we welcome you to schedule a meeting through PPMD For You.

The afternoon focused on quality of life, and PPMD’s Ellen Wagner moderated a conversation among attendees, specifically how to build support systems for your entire family. Isabella Servi shared her perspective as a sibling growing up with a brother living with Duchenne, and Jill Castle, M.Ed discussed how she cultivated her village caring for her son over the years.

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

Duchenne 101 Panel

PPMD’s Amanda Wilkison, RN, BSN discussed educational care materials that are available to families free of charge including our THINK emergency resources, comprehensive guides, and more.

PPMD’s Kayla Quirin, MS, CGC presented a genetics overview with information about joining The Duchenne Registry and PPMD’s free genetic testing program, Decode Duchenne.

Dr. Bridget McGowan provided and overview about research participation and elements to consider before entering a trial.

Jill Castle, M.Ed spoke about tips and tricks for school planning, her experience advocating in the school setting, and creative ways to overcome barriers.

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

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

Italfarmaco

Learn more about Italfarmaco’s investigational therapy, Givinostat, an HDAC inhibitor, with a review of results from their completed Phase 3 study

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

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