ENERGY IN ACTION - EPISODE 029 AllStripes & Cyclerion - Community Collaboration to Push Clinical Trials Forward Richard Elles, Director of Patient Advocacy & Industry Engagement with AllStripes and Chad Glasser, Director of Clinical Research with Cyclerion Therapeutics, join us to discuss a pivotal clinical trial that Cyclerion Therapeutics has and the collaboration we have with AllStripes to help move the research initiative forward. EPISODE HIGHLIGHTS Can you tell us about Cyclerion's MELAS Study? Cyclerion Therapeutics has an ongoing clinical study in patients with Mitochondrial Encephalopathy, Lactic Acidosis, and Stroke-like episodes (MELAS) that's evaluating the investigational drug 6463. The study is a 29 day study and looks at safety and tolerability primarily and also determining what 6463 does to the disease. Next steps are subsequent confirmatory studies to be conducted, further confirming safety and efficacy and to move closer to an eventual approval. What is the importance of patient participation in clinical trials? Drugs can't be approved and the safety and efficacy of drugs can't be evaluated without patient engagement. We have a responsibility to study drugs in a sufficient number of patients to fully understand effects and that's how approval is justified and moves through phases of development. What would you like qualified patients to know about participating in the trial? We know a lot about this molecule and we feel confident in the safety profile and efficacy. This study has been designed to be as easy and feasible as possible. Things like remote visits and travel reimbursement are offered to make it more convenient for patients to participate. What does AllStripes do and how does that work compliment pharmaceutical work? The mission of AllStripes is to unlock ...
PARENTS AS RARE - EPISODE 052 Lisa Weinberger - Be Your Own Advocate, Listen To Your Body, Take Control of Your Health, & Ask Questions Lisa Weinberger is a wife, mother, digital marketing professor and business owner with over 20 years of experience designing and leading corporate marketing programs. We talk in this episode about balancing work and family while living in the world of rare disease and chronic illness. EPISODE HIGHLIGHTS When you were navigating a diagnosis, how did that affect your family at the time? I was diagnosed with a rare disease called Pemphigus Vulgaris in 2018 after 30 years of varying symptoms. My disease rarely showed physical symptoms and I wasn't open with my daughter or in-laws who lived just down the street. Around the same time that I began getting blisters and was concerned about hiding them from my daughter, my husband had a heart attack which captured her attention more. Afterwards, I spoke with her about what was going on, explaining my auto-immune disease and the symptoms I was experiencing. When things are especially challenging because of your disease, how do you push through the days? I have a glimmer of hope and I know I've been in similar situations before. I am a fighter and have a mentality to keep going. I push myself and I'll continue to push myself with time to rest as needed, but I do tend to overdo it and not pace myself during the times I feel the best. How do you adjust your life as a result of your circumstances and keep up with your family's activities? My husband did a lot when my daughter was younger, ...
ENERGY IN ACTION - EPISODE 064 Lovevery - Purposeful Play Customized for all Abilities Maral Amani is a Licensed Physical Therapist and the Disability Support Specialist at Lovevery, a toy company that helps families, educators and therapists find the right toys and the right tools to help develop confidence through play. EPISODE HIGHLIGHTS Can you tell us about yourself and your work? I'm a Physical Therapist, specializing in children ages 0-3 and early intervention. At Lovevery, I'm the Disability Support Specialist for a recently launched program that allows families to purchase play kits without a subscription. Through communicating with me, I'm able to help families choose specific play kits that are best for children with a focus on skill-based development and strength-based development. Families can reach out to me through the website, answer questions about their children, share what skills they're developing, and I will recommend a play kit that will also include a play guide. What kits are available for children with mobility issues? As a Physical Therapist, I focus on big movements, core strengthening, leg strengthening, bearing weight through the arms. For tiny movements, it's best to put children in a comfortable position to play so they're reducing the energy demand of holding themselves up so their energy can focus on fine motor movement. If you purchase a play kit that has toys that can't be used right away or used in the typical way, think about how the toy can be enjoyed with modifications. What do you recommend for developing executive functioning skills? My favorite play kit for that is the Inspector Kit with the ball drop box. It comes with balls inside the box, but ...
