Signup to receive the UMDF newsletter in your email monthly at umdf.org/newsletter-signup.
For the first time, the testing is being offered on UMDF’s patient registry, mitoSHARE, enabling a seamless transition for genetic testing participants to receive curated results, connect their data to share with a clinician, participate in surveys and studies, and more. The program is once again in partnership with Probably Genetic, which utilizes an online symptom assessment to determine testing eligibility. The test, administered by Variantyx, is based on a whole genome backbone and will return results for all known pathogenic nuclear genes associated with mitochondrial disease as well as full mitochondrial DNA sequencing. Patients can find instructions on how to explore their eligibility by visiting umdf.org/genetictesting. Join mitoSHARE at umdf.org/mitoSHARE. Email registry@umdf.org with questions. This program is supported in part by an unrestricted grant from UCB. |
||||||||||||||||||||||||||
|
Affected patients and families are being asked to take a needs assessment survey to help UMDF shape the future of the organization’s programming. This new survey, which should take 15-20 minutes to complete, is available now at umdf.org/patient-needs-assessment. “We hope these questions will help identify community priorities, show where we have unmet needs, and ensure that our programs and resources remain relevant to everyone we serve,” said Margaret Moore, UMDF’s Associate Director of Support and Education. |
||||||||||||||||||||||||||
|
We’re excited to unite with other mitochondrial disease-focused patient advocacy groups across the globe to spread awareness on World Mitochondrial Disease Week, Monday, Sept. 15 to Sunday, Sept. 21! Together, we can promote understanding and recognition with the hope that increased awareness, attention, and funds will move the needle toward effective treatments and cures for the entire mito community.
Stay tuned to our World Mitochondrial Disease Week 2025 page for all the ways you can get involved. |
||||||||||||||||||||||||||
|
Last week, Barth Syndrome Foundation (BSF), UMDF, and MitoAction leadership were notified by Stealth BioTherapeutics that the FDA has refused their request to reconsider the elamipretide new drug application (NDA). This refusal to reconsider comes despite a positive Type A meeting where FDA reviewers signaled that Stealth should proceed with a formal request for reconsideration. Instead, the FDA has requested that Stealth BioTherapeutics resubmit their NDA, for the third time, using the accelerated approval pathway but under the longer of two resubmission timeline options. The means the process, already more than 18 months long, will be extended another six months, plus another 30 days for FDA review. While Stealth is still offering elamipretide via an Expanded Access Program (EAP) today, this additional delay is likely to affect EAP access for all mito patients, not just Barth syndrome families – and ultimately could mean the end of the company and drug before the results of the NuPOWER Phase 3 clinical trial for primary mitochondrial myopathy can even be considered. We, together with BSF and MitoAction, are doing everything we can to change this trajectory. A letter to the FDA, cosigned by more than 70 clinicians serving mito patients, asking for a final reconsideration, or at a bare minimum, a shorter accelerated approval timeline was submitted to the FDA this week. We encourage you to visit our UMDF Advocacy Center, which lays out the steps you can take to help. |
||||||||||||||||||||||||||
|
UMDF’s Ask the Mito Doc series returns this September with a webinar focused on the management of seizures in mitochondrial disease patients on Tuesday, September 16, at 7pm ET. Speakers include: Russell Saneto, DO, PhD, Seattle Children's Hospital, Seattle, WA Click here to register or here to submit questions beforehand. Bench-to-Bedside Seminar for September Researchers and clinicians, join us on September 8 at 12pm ET for a Bench-to-Bedside Seminar focused on The Role of Rehabilitation Medicine in the Care of Children with Mitochondrial Disease. Click here to register. Speakers include:
|
||||||||||||||||||||||||||
|
More than a dozen Energy for Life walks are coming to cities across America this summer and fall. Energy for Life events help fund mitochondrial disease research, education, and advocacy, while supporting and connecting the mito community. Last year, such walks 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 a planning committee for an Energy for Life event! Get involved by visiting energyforlifewalk.org.
|
||||||||||||||||||||||||||
|
MitoWorld Talks Mito in Space, Mito & Cancer, and More Each month, UMDF partners with MitoWorld for #BeyondTheDisease, a feature highlighting advances in mitochondrial science and the people responsible for them. This month, read multiple features on mitochondria in space with researchers Chris Mason, PhD, of Weill Cornell Medicine, and Afshin Beheshti, PhD, of University of Pittsburgh; how cancer cells benefit from the transfer of energy-producing mitochondria obtained from neuron; and a profile on the Functional Genetics of the Oxidative Phosphorylation System group at the Spanish National Centre for Cardiovascular Research. |
||||||||||||||||||||||||||
|
As a volunteer-driven organization, much of what you see happening at UMDF involves a dedicated army of volunteers who generously provide their time and talent to help move UMDF’s mission forward. You're invited to join us via Zoom on Wednesday, Sept. 3, from 12:00 pm - 1:00 pm ET for a Volunteer Interest Meeting 2025 where you can find out more about volunteering at UMDF, including opportunities like becoming a support ambassador, advocate, or fundraiser. Please register here. |
||||||||||||||||||||||||||
|
Highlighted Support Events This Month August 13, 12:00 pm - 1:00 pm ET, Support Group for All August 18, 8:00 pm - 9:00 pm, Parent and Caregiver Virtual Support Meeting August 26, 12:00 pm - 1:30 pm ET, Mindfulness with Mary Series September 4, 8:00 pm - 9:00 pm ET, Adult Virtual Support Meeting September 16, 7:00 pm-8:00 pm ET, UMDF Ask the Mito Doc |
||||||||||||||||||||||||||
UMDF Launches New No-Cost Genetic Testing Program via mitoSHARE
