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UMDF Launches New Clinical Trials Finder
Beyond the online portal, for the first time ever, a “concierge service” – guides known as clinical trial navigators – will be available to assist mitochondrial disease patients, families, and clinicians via phone at (844) 893-5528. “We continue to hear from families that interpreting eligibility criteria is a major barrier to clinical trial enrollment,” said UMDF’s President & CEO Kristen Clifford. “We’re extremely excited to launch this new platform, which we think will ease many of the problem points when families look for trials.” |
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UCB Announces FDA Acceptance of New Drug Application for Potential TK2d Therapy
“This announcement from UCB further underscores our belief that this community is on the precipice of incredible things,” said UMDF President and CEO Kristen Clifford. “Just a decade ago, a therapy for a devastating mitochondrial disease like TK2d would have been unthinkable. We thank the army of researchers, clinicians, patients, and caregivers who so selflessly helped us reach this point, along with UCB for their steadfast commitment to shepherding this drug to where we stand.” With the announcement, there are now three PDUFA action dates related to potential mitochondrial disease therapies scheduled for 2025, including UCB, Stealth’s elamipretide focused on Barth Syndrome (action date April 27), and Saol Therapeutics’ sodium dichloroacetate oral solution focused on Pyruvate Dehydrogenase Complex Deficiency (action date May 27). |
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Join us for the Mitochondrial Medicine 2025 Masterclass on Primary Mitochondrial Myopathy with a Focus on TK2d
The Masterclass, which will be Saturday, June 21 from 9am – 2pm, will spend a day going over Primary Mitochondrial Myopathy with a Focus on TK2d, with two tracks -- Early Career Investigators and Up-and-Coming Clinicians. The Up-and-Coming Clinician track will sharpen clinical skills, expand knowledge of Thymidine kinase 2 deficiency (TK2d), a rare genetic mitochondrial disease, and primary mitochondrial myopathies, and help with the tools to provide better, more personalized care for patients. Whether you’re a budding clinician or seeking to specialize further, this is the training you don’t want to miss. The Early Career Investigator track is tailored to meet the unique needs of early career investigators in mitochondrial medicine, providing expert insights, valuable networking opportunities, and practical strategies for success in academia and industry. Whether you’re just starting your career or seeking to build upon your existing experience, these sessions will help you chart your course for long-term success. The program is included in the standard Mitochondrial Medicine SciMed registration or can be purchased for the single day. Scholarships are available. For more information, visit the Masterclass page. The Masterclass is funded via a medical education grant by UCB.
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Energy for Life Walks Hit Pittsburgh, St. Louis,
Energy for Life Walkathons help fund mitochondrial disease research, education, and advocacy, while supporting and connecting mito families. Last year, walkathons brought in over $500,000 from across the country, money that is crucial in helping chart the course toward mitochondrial disease treatments and eventual cures. If you or someone you know is interested in doing more for the mito community, contact us to learn how you can be a part of the planning committee for an Energy for Life event! Make a difference today!
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UMDF Advocacy |
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UMDF & Friends on the Road  Attending MDA Conference March 16-19 in Dallas? Look for the UMDF at our booth in the patient advocacy pavilion, booth #2, and our mitoSHARE abstract with the posters, #P249.UMDF Support Ambassador Daniel Barsook will be a panelist at the The Mitochondria in Health and Disease Gordon Research Conference on March 23 - 28, 2025 at the Ventura Beach Marriott. The conference is focused on advancing the frontiers of science through the presentation of cutting-edge and unpublished research, prioritizing time for discussion after each talk and fostering informal interactions among scientists of all career stages. The conference program includes a diverse range of speakers and discussion leaders from institutions and organizations worldwide, concentrating on the latest developments in the field. Click here for more information.
(Note this conference has specific education / background requirements) UMDF’s Science and Alliance Officer Dr. Philip Yeske will be a panelist at the Mitochondrial Transplantation and Next Generation Therapeutics Conference on April 27 - 29, 2025 at Hofstra University on Long Island. The conference will explore the fascinating and rapidly evolving field of mitochondrial transfer and transplantation. Engage, connect, and collaborate with mitochondrial transfer and transplantation experts, industry researchers, and patient advocacy groups on approaches for developing novel therapies and the latest advancements and breakthroughs. Click here for more information.
The Department of Genetics and Genomic Sciences at the Icahn School of Medicine at Mount Sinai invites mitochondrial disease families to join them on March 12 -- in-person or remotely -- for Frontiers in Mitochondrial Medicine, a symposium on the Mount Sinai Campus in the Upper East Side in Manhattan showcasing cutting-edge research and breakthroughs in mitochondrial biology and therapeutics. Featuring an illustrious lineup of leading experts and a keynote lecture by Dr. Douglas Wallace, this event will highlight emerging insights and innovative approaches transforming the field. Programming is expected to start at 11:30am ET. Click here to register.
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#BeyondTheDisease: A New Form of Mitochondria, mtDNA, Diabetes, and more
This month, the MitoWorld team talks with Dr. Craig Thompson of Memorial Sloan Kettering Cancer Center about his team’s discovery of a new form of mitochondria and interviews Dr. Carlos Moraes about experiments to affect mitochondrial DNA (mtDNA). Plus, MitoWorld editor Gary Howard reviews research surrounding the connection between diabetes and mitochondria and examines the supporting interactions of the organelles and messengers that support mitochondrial functions. |
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Clinical Trial Readiness and Mito Med Clinical Research Pavilion Update Focus of March's Combined Ask the Mito Doc and Bench-to-Bedside Speakers include:
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New Features Coming to mitoSHARE
mitoSHARE is a worldwide research database for patients (both living and deceased) with any form of mitochondrial disease, as well as their caregivers. A confirmed diagnosis of mitochondrial disease is not required to join mitoSHARE. Patients at any stage of the “diagnostic journey” are encouraged to register. If you’ve been thinking about joining mitoSHARE, now is a great time with so much on the horizon. Join today at umdf.org/mitoSHARE. |
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Highlighted Support Events March 10, 12:00 pm - 1:00 pm ET, UMDF Ask the Mito Doc Webcast Series – Clinical Trial Readiness and UMDF Mito Med Clinical Research Pavilion Update March 17, 8:00 pm - 9:00 pm ET, Parent and Caregiver Virtual Support Meeting March 25, 12:00 pm - 1:30 pm ET, Mindfulness with Mary Meditation Series April 3, 8:00 pm - 9:00 pm ET, Adult Virtual Support Meeting April 9, 12:00 pm - 1:00 pm ET, Support Group for All |
As part of the organization’s commitment to a “culture of clinical trials,” UMDF has partnered with Carebox to launch a new clinical trials finder tool focused on mitochondrial disease. You can access the tool on 
Last week, UCB announced that their New Drug Application for doxecitine and doxribtimine for the treatment of Thymidine Kinase 2 deficiency (TK2d) has been accepted by the FDA. While no official PDUFA action date was included, the announcement noted it has been granted priority review, meaning an answer could come as early as August 2025. 
For the first time ever, Mitochondrial Medicine Conference will be hosting a Masterclass for the science and medical community.
Mito Med 2025 News
This spring’s Energy for Life events in 
The new administration and Congressional leaders are suggesting major changes to Medicaid to reduce overall federal spending. As part of UMDF’s commitment to representing the needs of the mitochondrial disease community on Capitol Hill, we're asking for your feedback via the survey link below so we can share how potential changes would impact your family. 
Each month, the research team at UMDF partner, 
UMDF’s Bench-to-Bedside and the Ask the Mito Doc Webcasts are combining this month to focus on Clinical Trial Readiness. Instead of two separate webcasts, we will combine the clinician Bench-to-Bedside with the patient/family focused Ask the Mito Doc into ONE webcast. The combined webcast will take place from 12:00-1:00PM ET on March 10th. The audience will be a mix of patients, researchers, clinicians and industry. Amongst the topics are Clinical Trial Readiness, along with UMDF Mitochondrial Medicine Conference Clinical Research Pavilion (CRP) 2024 readouts and preview of what to expect in the CRP in St. Louis this June. 
Even more exciting features are coming to UMDF’s patient registry, mitoSHARE, this spring, including new ways to participate in research, biorepository options, and more. With all these new options, we’ll be adjusting the consent form – so participants should be on the lookout explaining new informed consent requirements.