KrabbeConnect YouTube Videos

About the Webinar (Recorded on September 26th, 2024)
This session will provide an in-depth look at the latest findings from these pivotal trials focused on treatments for Krabbe disease. Attendees will gain valuable knowledge on trial outcomes, patient responses, and the potential implications for future therapies. Whether you’re a researcher, clinician, or someone impacted by Krabbe Disease, this webinar offers critical updates and data-driven insights into these groundbreaking studies.

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This panel was recorded on September 16th, 2024. The panel, moderated by Stacy Pike-Langenfeld, explores various experiences, showcasing the challenges and triumphs in the treatment of Krabbe disease. Each caregiver will talk about their child’s journey. Each journey is unique, giving healthcare providers and newly diagnosed families "insider knowledge," of what that first year could be like after a Krabbe disease diagnosis.

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A Krabbe disease study led by Dr. Nicholas Bascou. Dr. Bascou completed his undergraduate studies at the University of Pittsburgh at The Program for the Study of Neurodevelopment in Rare Disorders (NDRD). He then obtained his M.D. at the Cooper Medical School of Rowan University in Camden, New Jersey, and completed his training in internal medicine at Albert Einstein Medical Center in Philadelphia, PA. Dr. Bascou, a physician resident in Neurology at Johns Hopkins University states, "It is incredible to see the leaps in our understanding of Krabbe disease over the past decade and the new treatments that are becoming available. Our team is extremely grateful to RFRF for providing the resources needed to help conduct a study that we hope will demonstrate how big of an impact NBS and disease-modifying therapies have on the lives of patients and their families affected by Krabbe."

The study intends to gather valuable insights through parent-reported data, enabling a comprehensive assessment of the challenges faced by individuals living with Krabbe disease. By comparing the quality of life among patients who have received transplants and those who haven't, the research aims to shed light on potential differences in outcomes, informing future treatment strategies, patient care, and most importantly, the importance of early intervention.

We invite you to take 15 minutes to learn more! We encourage everyone to take the initial survey whether you qualify or not! This allows us to gather insights on the current Krabbe disease demographics. Those who qualify for the study will be asked to participate in a 60-90 minute interview. Start your survey here- https://www.engagehealth.com/survey/LoginCode.aspx?SurveyID=8lKJ6l53

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Meet the Moore sisters. Raegan missed the window of opportunity for a potentially life-saving intervention because her formal diagnosis did not arrive until she was 9 months old. Raegan faced the heartbreaking challenges of severe neurological deterioration, loss of mobility, and a shortened life expectancy.

Meet Zoey, a joyful and energetic young adult fortunate to be diagnosed with Krabbe disease shortly after birth because of her sister. Early detection through newborn screening allowed her immediate medical intervention. Zoey received a life-saving stem cell transplantTypically this refers to a bone marrow transplant with the goal of replacing non-working cells with healthy working cells.  Bone marrow is a rich source of stem cells that have the unique ability to that halted the progression of the disease and significantly improved her prognosis. Thanks to the timely diagnosis and treatment, Zoey is thriving and enjoying a relatively normal life. Zoey's story underscores the importance of early detection and the positive impact it can have on children with Krabbe disease.

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This is the full heartwarming interview with Bruce Kirbo. Grandpa Bruce went to work on Cove's Law after his grandson, Cloud, was diagnosed with Krabbe disease. Listen to how Bruce, and other advocates, successfully launched a pilot study in Georgia for Krabbe disease.

Krabbe disease (pronounced krab A) is a rare genetic disorder known as globoid cell leukodystrophyAnother name for Krabbe disease, a rare and usually fatal disorder of the nervous system.. Krabbe disease is a severe neurodegenerative and rapidly progressing condition requiring immediate treatment for the most severe forms. A delayed diagnosis, especially in the most severe forms, equates to palliative and supportive care as the only means of treatment until premature death.

We invite you to learn more about this disease and all the heroes impacted by this neurological disease at https://krabbeconnect.org/

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This is the uncut video from our interview with the Kirbo family. Learn about all of the emotions involved in navigating Krabbe disease through the eyes of the Kirbo family. KrabbeConnect is forever grateful to Carlyle and Sloane for sharing their son's story to help further education and improve awareness about the need for additional therapies for Krabbe disease.

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Bruce Kirbo went to work on Cove's Law after his grandson, Cloud, was diagnosed with Krabbe disease. Listen to how Bruce, and other advocates, successfully launched a pilot study in Georgia for Krabbe disease.

Krabbe disease (pronounced krab A), is a rare genetic disorder, also known as globoid cell leukodystrophyThe leukodystrophies comprise a group of progressive, genetic disorders mainly affecting the central nervous system (CNS). Most leukodystophies result from a disruption of the growth of the myelin sh. Krabbe disease is a severe neurodegenerative and rapidly progressing condition requiring immediate treatment for the most severe forms. A delayed diagnosis, especially in the most severe forms, equates to palliative and supportive care as the only means of treatment until premature death.

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We invite you to learn about the Kirbo and Ellis families. Both have been impacted by Krabbe disease. Cloud has a late-onset form of Krabbe disease and Cove has the infantile form of the disease. Cloud and Cove offer so much to their families despite their disabilities. Both children are surrounded by love and their families hold out hope for additional therapies to help further treat Krabbe disease. We invite you to learn more about Krabbe disease KrabbeConnect.org

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There’s no shortage of talent in the science and clinical space of Krabbe disease. KrabbeConnect believes it’s vital to honor an individual every year who has made a significant contribution(s) to Krabbe disease in one or more of the following areas: educating and improving methods to effectively recognize and diagnose Krabbe disease, advancing breakthrough research into novel treatment(s), andor improving the quality of life for patients through innovative research and clinical interventions.

Dr. Joanne Kurtzberg is an internationally renowned expert in pediatric hematologyoncology, pediatric blood and marrow transplantation, umbilical cord blood banking and transplantation, and novel applications of cord blood in the emerging fields of cellular therapies and regenerative medicine. Since 1990 she has served as the director of the Pediatric Blood and Marrow Transplant (PBMT) Program at Duke University Medical Center. The PBMT program has transplanted over 60 patients affected by Krabbe Disease. Dr. Kurtzberg's current research is focused on finding ways to use cord blood cells to help children with Krabbe disease and other leukodystrophies. To learn more about Dr. Joanne Kurtzberg please visit Duke University Medical Center.

We invite you to learn about the impact of her work at Duke University Medical Center from this impact video.

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Are you ready for the #curekrabbbe challenge? The Krabbe community of patients and their caregivers are challenged each day due to their Krabbe disease diagnosis. KrabbeConnect works to help families with these challenges by advancing research, providing family support, enhancing education, and raising awareness. KrabbeConnect believes in working together to help assure a better life for Krabbe disease patients yet, we cannot continue our work without your help! Please join us to #curekrabbe in the Portland to Portland 2022 Challenge. Visit KrabbeConnect by April 14th, 2022 to register.

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This video debuted at the KrabbeConnect 2021, A Million Dreams Gala. Learn about Jasper who was in foster care, was identified with Krabbe disease as a newborn, and how he's doing today. The Murphy family is thankful for the love and joy Jasper brings to their home.

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This video was first debuted at the 2021 KrabbeConnect, "A Million Dreams," gala. Dr. Allison Bradbury has evaluated disease mechanisms and developed gene therapyA type of therapy that offers hope and promise for a cure for many genetic disorders.  A working copy of the gene replaces the non-working copy of the gene. Gene therapy is at the forefront of many approaches for Krabbe disease. The Bradbury Laboratory is dedicated to understanding disease mechanisms and developing therapeutic approaches for rare, pediatric neurodegenerative disorders. Advances in adeno-associated virus (AAV) vectors have led to safer and more efficient viral vehicles to deliver therapeutic transgenes in a single injection, and gene therapy is now a favorable therapeutic intervention for monogenic diseases.

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one marrow transplants are utilized in patients diagnosed with Globoid Cell Leukodystrophy (Krabbe) to help treat the debilitating disease. However, some of the chemotherapy medicines utilized to suppress the immune response can leave a patient managing troublesome side effects. Thus, transplant specialists are pioneering a new way to conduct bone marrow transplants in patients with non-malignant disorders such as Krabbe disease. Dr. Paul Szabolcs will present his recent publication discussing the use of RIC in patients with Krabbe disease. Dr. Paul Szabolcs is the Division Director at the Blood and Marrow Transplantation and Cellular Therapies at the University of Pittsburgh.

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Did you miss the gala on Friday, September 24th, 2021? No problem, you can view it here now.

KrabbeConnect is truly thankful to be here, providing those directly living with Krabbe disease educational resources to make difficult decisions a bit easier, connecting them to rare disease experts, and participating in research efforts to help #curekrabbe. We hope you feel informed and inspired by this community working to improve education, awareness, and research for Krabbe disease. We invite you to consider a donation (https://krabbeconnect.org/donate/) to help advance our mission and work at KrabbeConnect.

Warm Regards-

KrabbeConnect Board of Directors

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This video was utilized for the 2020, "A Million Dreams," Gala. A great story of a family impacted by infantile Krabbe disease. If this story moves you, help us reach a cure by donating at KrabbeConnect.org today.

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