by: Parent Project Muscular Dystrophy
End Duchenne Tour 2020: Virtual Tour Stop in St. Louis, MO
On Saturday, October 17, we spent the morning with the Duchenne community in St. Louis, MO and the surrounding region for another End Duchenne [Remote] Tour stop. We were joined by 12 families, 12 healthcare providers, and 8 industry partners. Although we wish we could have met in-person, we were happy to connect virtually and share updates around care, research, genetics, and how to get involved.
This event included a unique opportunity to participate in both pediatric and adult care breakouts. The pediatric care breakout was moderated by PPMD’s Rachel Schrader, and included key members of the Children’s Mercy Kansas City neuromuscular team. The adult care breakout was moderated by PPMD’s Amanda Wilkison, and included members of the University of Missouri – Columbia team. We are so grateful to have both of these amazing teams as PPMD Certified Duchenne Care Centers to provide excellent multidisciplinary care and support to families in Missouri and the surrounding region.
SNAPSHOT OF THE DAY
Our day began with PPMD’s SVP of Clinical Care, Rachel Schrader, providing historical background and current key updates on PPMD’s research, advocacy, and care programs.
Download PDF >JOIN THE FIGHT TO END DUCHENNE
We were joined by Kate VanderWeele and Jess Divin, parents and Connect Coordinators of PPMD’s Connect Illinois. Kate spoke about the importance of establishing local support and getting involved. Although there is not yet a PPMD Connect group in Missouri or Kansas, Kate and Jess encourage families to contact them directly or join the PPMD Connect Illinois Facebook Group to learn more about ways to connect with others. If you or someone you know is interested in starting a group, please contact Nicole Herring.
We also heard from Abbie Killian, who is a sibling of someone living with Duchenne. She is one of the original founders of PPMD’s new Sibs Connect group. To learn how siblings can get involved, email Abbie directly, join their Facebook Group or respond to the interest survey on the PPMD website.
THE DUCHENNE REGISTRY
We were joined by PPMD’s Niki Armstrong, MS, CGC, one of PPMD’s Genetic Counselors. Niki provided background on the genetic implications of Duchenne, as well as the importance of genetic testing, including PPMD’s free genetic testing program, Decode Duchenne. Niki also emphasized the value of joining the Duchenne Registry to become a citizen scientist to help advance the field.
Download PDF >PARTNER INTERACTIVE DISCUSSION
Rachel invited our industry partners to participate in a panel discussion. We want to thank representatives from PTC, Sarepta, Santhera, Pfizer, and Italfarmaco for spending their morning with us and answering questions from the community.
We are also so thankful to our partners that supported this Tour stop and our efforts this year. Please click here to view information regarding current sponsor trials and a research overview, and submit questions at any time.
View Presentations >PEDIATRIC INTERACTIVE CARE PANEL
Rachel moderated the pediatric care panel breakout and facilitated discussion between attendees and members of the Children’s Mercy Kansas City team, one of PPMD’s Certified Duchenne Care Centers (CDCCs). We want to Dr. Ann Modrcin (PM&R), Alexis Hazlett (Care Coordinator), Stephanie Orr (Physical Therapist), Dr. Nikki Singh (Cardiologist), and Dr. Samira Naime (Pulmonologist) for participating.
- Dr. Modrcin and Alexis spoke about the importance of multidisciplinary and coordinated neuromuscular care that should include a plan to transition to adult care
- Stephanie talked about physical therapy evaluation, development of a plan of care, and recommendations for individual needs in Duchenne
- Dr. Singh spoke about cardiac care in Duchenne and considerations about starting cardiac medications early to delay or slow down cardiac changes
- Dr. Naime discussed respiratory management in Duchenne including the importance of regular PFTs, screening for sleep-related problems, and receiving scheduled vaccines and annual flu shots.
ADULT INTERACTIVE CARE PANEL
Amanda moderated the adult care panel breakout and facilitated discussion between attendees and members of the University of Missouri – Columbia team, PPMD’s first Adult Certified Duchenne Care Center (CDCC). We want to Dr. Raghav Govindarajan (Neurologist), Dr. Vovanti Jones (PM&R), Dr. Jonathan Ang (Pulmonologist), and Dr. Vanessa Kuwajima (Gastroenterologist) for speaking.
- Dr. Govindarajan & Dr. Jones spoke about the multidisciplinary team at University of Missouri, and how the roles of neurology and physical medicine & rehabilitation succinctly work together to provide optimal neuromuscular care
- Dr. Ang talked about the importance of monitoring lung muscle function through PFTs, Peak Cough Flow, and Sleep studies and how to manage issues with assisted cough and lung volume recruitment (i.e. assisted ventilation)
- Dr. Kuwajima discussed her role in the neuromuscular team to manage issues with digestion including motility issues, constipation, and supplemental nutrition
NEXT UP
The final End Duchenne [Remote] Tour stop in 2020 will be in Miami, FL on Saturday, December 5th and will include bilingual presentations for both English-speaking and Spanish-speaking families. This virtual meeting will take place on Saturday, December 5th.
Though nothing is normal about our new, we so appreciate the time we spend with you and please know we are here to support you however we can.
