Cure LBSL

This past weekend, the team I created of Neuroscience Research Scientists at Kennedy Krieger Institute participated in the Baltimore Running Festival, running a 10k and successfully raising over $1,000! We are incredibly proud and honored to have been part of this impactful initiative. During the event, we proudly sported our #TeamKennedyKrieger T-shirts and #CureLBSL body paint. #NeuroscienceResearch #KennedyKrieger #BaltimoreRunningFestival Team: Bayley Lindsay, Christina Nemeth Mertz, Chase Solomon, Joseph Mertz

At #CGIC24 this morning, a powerful panel titled "Lived Experiences in Action: Advocacy Updates Across Rare Diseases" highlighted the vital role of patient voices in drug development. C-Path, alongside regulatory bodies like the FDA and European Medicines Agency, and its consortia members and partners, is fully committed to ensuring that patients are at the center of every step in the process, truly walking the talk in rare disease advocacy. Michelle Campbell (FDA): "Patient Focused Drug Development is a systematic approach to help ensure that the patients experience, perspectives, and needs as well as requirements are being captured in a meaningful way and are being incorporated in drug development and evaluation of treatments...it has allowed us to capture this experience throughout the drug development cycle."   Andrea Pauls Backman (ALS Strategy Consulting): "I am excited because we are at a real reflection period with ALS, we've had a lot of trials and we need to understand patient voice, and it's not a singular voice, but patient voices throughout the process."   Stacy B. Lewin, MD (ALS Advocate): "An average life span for an ALS patient is 2-5 years. It is not a lot of time to be involved in research. My being involved in research trials as a patient is not for me, it's for the future." "It is so important to get the patient voice. As you can see from Layne and I, this is very challenging, but we want to use our voices to make trials for future treatment options, and more accessible for patients."   Layne Oliff (ALS Advocate): "I'm not a big fan of the words "lived experience" when we talk about patients with ALS. I'm not dead though, I am living. I am living experience not LIVED." The focus should be on research and finding a Cure!"   BJ Viau (HD Youth Organization): "We need to have more conversations like this [with patients]. This has given us the opportunity to accelerate treatments, accelerate policy with patient communities." Thomas H. Greg Pruitt Spastic Paraplegia Foundation, Inc   #CPath #PatientVoice #DrugDevelopment #RareDisease #drugdevelopment #datasharing #globalimpact

This weekend I had the privilege of presenting my research from the Fatemi lab in our ongoing efforts to develop a potential therapeutic for LBSL at the 2024 LBSL Patient and Scientific Conference. Thank you to the incredible LBSL community, Cure LBSL, and the Kennedy Krieger Institute for curating such an wonderful event. It is humbling to be even a small contributor to such an inspiring group. #TimeIsMyelin Inés Garofolo Christina Nemeth Mertz

I love representing Kennedy Krieger Institute and discussing our research on LBSL! It's an honor to share some key moments with Bayley Lindsay, Christina Nemeth Mertz, Michael McGinn, and Melody Kisor. Take a look at https://lnkd.in/eYj7Y7cy to learn more!