Evenement flyer

BOKS viert zijn 25e verjaardag

Ter gelegenheid van het 25-jarig jubileum organiseert BOKS een evenement met de naam ‘BOKS connects! Het belang van samenwerking '. Het evenement zal experts op het gebied van mitochondriale ziekte en belangenbehartiging van patiënten bijeenbrengen.

 

 

 

 Evenement flyer

Image of a model of a human heart

Elamipretide trial shows encouraging results in improving cardiac function in Barth syndrome patients. 

A Phase 2/3 TAZPOWER study evaluating elamipretide in patients with Barth syndrome (a type of mitochondrial disease) has shown encouraging results in the improvement of cardiac function.

"Based on the data presented showing an increase in stroke volume, treatment with elamipretide appears to have improved heart function, which might indicate cardiac remodelling," noted Dr. W. Reid Thompson, Associate Professor of Pediatrics at the Johns Hopkins University School of Medicine. "Most patients with Barth syndrome have underlying heart disease, so a cardiac effect would be an important outcome in this setting that warrants further investigation."

Other elamipretide clinical trials focusing on different types of mito, including Primary Mitochondrial Myopathy are currently taking place.

Each clinical trial, brings us a step closer to effective treatments and hope for the future.

Read full press release

 

Photo by Robina Weermeijer on Unsplash

You are not alone 

People affected by mito sometimes feel isolated: geographically, emotionally and socially. With some forms of mito being desperately rare, it can be difficult to find others with the same condition.

IMP Member organisations have a variety of support services for people living locally.

Where there is no national body, people affected by mito can seek support from IMP.

 

IMP’s support services include:

Closed Facebook Group

The IMP Members Group is a virtual support group for everyone affected by mito. Members are spread around the world, have many different types of mitochondrial disease, some are patients, others are parents and carers, some may have recently heard of mito and be at the start of their journey, others have been part of the mito community for years. 

Members offer support, share experiences and provide a safe space to connect with others affected by mito.

If you’d like to connect with others in the mito community, join us today.

WHEREVER YOU ARE, YOU ARE WELCOME!

 

Medical Professionals Directory

We are building a directory of experts in mitochondrial disease who are willing to be contacted by people from countries with little or no specialist care. Contact us to request assistance with being put in touch with a medical professional.

If you are a clinician and would like to help mito patients in countries where local specialist medical assistance is limited or unavailable, please contact us.

 

Facebook page

Follow the IMP Facebook page to find out about the latest research and activities from around the world.

 

Contact us

Got a question? Get in touch via the webform, This email address is being protected from spambots. You need JavaScript enabled to view it. or Facebook and we’ll do our best to answer it – or find some who can!

(Photo by Fernando @dearferdo on Unsplash)

 

Five of the world’s leading mitochondrial disease (mito) patient advocacy groups have announced they will together pledge USD$1 million to form and fund a patient-driven research network to fast-track treatments and potential therapies for Leigh syndrome. The Leigh Syndrome International Consortium will focus on improving diagnosis, therapeutic development and optimized patient care.

Leigh syndrome, a form of mitochondrial disease (mito), is a debilitating and potentially fatal genetic disorder that robs the body’s cells of the energy they need to function properly. 

The groups forming and funding this research network include; the Mito Foundation (Australia), the United Mitochondrial Disease Foundation (UMDF) and the People Against Leigh Syndrome (PALS) in the US, along with the UK’s Lily Foundation and Mitocon of Italy. 

Mito Foundation CEO, Sean Murray, says by working together the groups are accelerating potential treatments for Leigh syndrome patients who have significant unmet medical needs. 

“Leigh syndrome is an inherited neurometabolic disorder that affects the central nervous system,” Mr. Murray said. “This progressive disorder begins in infants between the ages of three months and two years, however the prognosis for those with the disease remains poor. “This innovative, multi-year project will fund vital research to help to improve diagnosis and establish global best practices of care for Leigh’s patients,” he said.

Brian T. Harman, President and CEO of UMDF, said by uniting together this network will drive more connections to help develop future treatments for Leigh syndrome patients. “No single organisation can take on a project of this scale alone. This collaboration between mitochondrial disease patient advocacy partners will help us leverage our existing connections to the patient community as well as elite international researchers,” Mr. Harman said.

Dr. Bruce M. Cohen, MD, FAAN, Pediatric Neurologist and Director of the NeuroDevelopmental Science Center at Akron Children’s Hospital, will lead the international scientific steering committee for the Leigh Syndrome International Consortium. “Initially, the network will focus on natural history data collection, outcome measure development and validation as well as pre-clinical data coordination,” Dr. Cohen said.

“Later this year, the steering committee plans to develop a “Request for Proposals” to support Leigh syndrome-focused grants.”

For more information about the Leigh Syndrome International Consortium visit www.leighsyndrome.org

 

 

Thank to the Prime Minister of Australia The Hon. Scott Morrison MP for supporting the Mito Foundation and the mito community, and for raising awareness during Global Mitochondrial Disease Awareness Week.

The Prime Minister is the Patron of the Mito Foundation in Australia and has been a supporter for many years. 

 

Watch the video message here

 

© 2019 Int. Mito Patients