Clinical Trials and Research
|
|
Reata Announces Top-Line Data from the Dose-Escalation Cohorts of the Phase 2 Motor Study of Omaveloxolone in Patients With Mitochondrial Myopathies
|
“We appreciate the support of MitoAction and the mitochondrial disease community throughout the development of omaveloxolone as a potential treatment for mitochondrial myopathies. The data from the submaximal test are encouraging, and we are continuing to work with experts in the mitochondrial disease field to determine the appropriate next steps for development," states Colin Meyer, M.D.Chief Medical Officer and Vice President, Product Development.
|
|
Stealth BioTherapeutics Completes Enrollment of Study for the Treatment of Barth Syndrome
|
Stealth BioTherapeutics (
Stealth), a clinical-stage biopharmaceutical company developing investigational therapeutics to treat mitochondrial dysfunction, today announced the completion of enrollment in the
TAZPOWER clinical trial, a phase 2/3, randomized, double-blind, placebo-controlled crossover study to evaluate the effects of daily treatment with elamipretide in 12 patients with genetically confirmed Barth syndrome. Barth syndrome is a rare and debilitating genetic mitochondrial disease that is estimated to affect one in 200,000 to 400,000 individuals worldwide. There are no treatments approved by the U.S. Food and Drug Administration (FDA).
|
|
Diagnostic Odyssey of Patients with Mitochondrial Disease
|
Objective: To document the complex “diagnostic odyssey” of patients with mitochondrial disease.
Methods: We analyzed data from 210 Rare Diseases Clinical Research Network Contact Registry participants who were patients with a biochemical deficiency or self-reported diagnosis of mitochondrial disease, or their caregivers.
A podcast with Dr. Michio Hirano and Professor Seamus (John) Thompson about the paper will be available from the Journal Neurology April 3rd at https://www.aan.com/rss/?event=feed&channel=1
|
|
Making a Difference for Mitochondrial Disease
|
|
26.2 miles for Thomas Schmid and Sandra Russell
|
|
Zachary O'Neill and Jonathan Russell will be running the 2018 Boston Marathon on Monday, April 16th.
|
|
Zach, Captain of Massachusetts Maritime Men's Soccer Team, is running and raising funds in honor of Thomas Schmid, 11-year-old Mito patient. Help Zach reach his goal by
donating to his fundraising page today!
|
|
Jonathan is proud to be raising awareness and funds for the mitochondrial disease community in honor of his mother, Sandra Russell, who passed away from mitochondrial disease in 2008.
Donate to support Jonathan's efforts and make a difference!
|
|
Mito Warrior, Sheridan Johnston, has partnered with Maine Hempworks to create a lip balm. Maine Hempworks believes in giving back to the community. Purchase the Vanilla Chai lip balm and Maine Hempworks will donate 100% of the profits to MitoAction.
|
|
MitoAction Programs and Resources
|
|
|
|
Join us on Friday, April 6, 2018 at Noon EST/9 a.m. PST as Dr. Richard Boles discusses "Spectrum Needs, a New Comprehensive Nutritional Therapy for Autism, Functional Conditions, and Mitochondrial Disease"
|
|
|
|
|
Matthew Harty Scholarship
|
The Matthew Harty Scholarship was established in 2015 to honor the life of Matthew Harty, a North Andover, MA, boy who passed away from mitochondrial disease just days after his 8th birthday.
The Matthew Harty Scholarship will be awarded to a high school senior or currently enrolled college student with a diagnosis of mitochondrial disease. The student may apply by completing the application and writing a one-page essay. The essay must include how the money will be used to either further their educational studies or how it may be used to lessen the burden on managing their affliction to achieve their educational goals.
Matthew, the son of Sarah and Paul Harty, was an amazing little boy whose legacy will live on through these scholarship recipients.
The deadline to apply for the Matthew Harty Scholarship is May 15, 2018.
|
|
First Friday of the month: Guest speaker (noon EST)
Second Friday: Newly diagnosed patients and parents (noon EST)
Third Friday: Support group for parents (noon EST)
Fourth Friday: Support group for adult patients and spouses (noon EST)
Quarterly (Tuesdays): Autism-Mito Task Force (12:30 EST)
To participate, call the toll-free teleconference number 1-866-414-2828, participant code 017921#
|
|
The
Marcel's Way Family Fund is a program that offers a helping hand in the way of direct financial support to those suffering from mitochondrial disease.
Born out of the mission of continuing to improve quality of life for all affected by Mito, the Marcel's Way Family Fund has the potential to change the lives of many children and families who simply cannot afford the full cost of necessity such as wheelchairs, adaptive equipment, time lost from work during long hospitalizations, and medicines.
|
|
Tuesday April 10th
North Andover Middle School
North Andover, MA.
Sunday, April 29th
Central Rock Gym
Worcester, MA.
Saturday, May 5th
Venus II Restaurant Restaurant and Sports Bar
Marshfield, MA.
Saturday, May 5th
Royale
Boston, MA.
|
Sunday, Sept. 16th
DCR's Mother's Rest
Boston, MA.
|
|
|
|
|
The SKR Derby Day Benefit for Mito is ONE month away!
|
Join us in one month for the premier Kentucky Derby party in Boston! MitoAction's
Sandra K. Russell Derby Day Benefit for Mito is set for Saturday, May 5 at Royale in Boston. The event starts at 4:30 p.m. and includes Southern fare, auctions, a men's bow tie contest, a ladies hat parade, and so much more.
The funds raised at the event will go toward the programs and resources MitoAction brings to the Mito community, including:
- patient advocacy
- summer camp for Mito kids
- new patient education
- materials for clinicians
- support groups, podcasts, webinars, and educational events
Thank you to our 2018 SKR Derby Day Sponsors!
|
|
|
|
|
|
|
