Councils

Patient Association Committee

Patient Association Committee

Research into the use of biomarkers as a reliable tool for rapid diagnosis of and as an outcome measure for a range of NMDs will clearly have major consequences for patients and their families. BIO-NMD therefore will need to work closely with patients’ organisations.

The Patient Association Committee (PAC) will be composed of representatives from patients’ associations that will advise the Steering Committee (SC). The PAC will attend SC meetings, will advise the SC on patients' needs and concerns and ensure appropriate dissemination of the project aim and progress to patients.

 

 

Anna Ambrosini



Fondazione Telethon is a major Italian non-profit foundation, raising and distributing funds to advance scientific research towards the cure for muscular dystrophy and other genetic diseases.

Anna Ambrosini (PhD in Pharmacology and Toxicology) has15 years experience in research laboratories in the field of neuroscience. Since 2001 she has been Research Program Manager for the Italian Fondazione Telethon, where she is responsible for developing programs in the field of inherited neuromuscular diseases.

She is highly involved at a national level in new initiatives developed with a number of Italian neuromuscular Patients’ Associations as part of an NMD Alliance and is coordinating the activity of the Italian NMD clinical network as a partner of TREAT-NMD.  

She is member of the Steering Committee for the International Trial on Steroids in DMD (FOR-DMD study).

Since April 2009 she has been the Chair of the Executive Committee for the European NeuroMuscular Centre (ENMC) Consortium, where she represents Fondazione Telethon and the Italian Muscular Dystrophy Association (UILDM).

 

 

Marita Pohlschmidt



The Muscular Dystrophy Campaign has funded high quality research into neuromuscular disorders for the last 50 years. They provide free care and support for babies, children and adults affected by muscle disease, fund world-class research to find treatments and cures, provide grants towards equipment and campaign to raise awareness and bring about change.

 

Marita Pohlschmidt is the Director of Research at the Muscular Dystrophy Campaign. She in charge of an annual £2m budget invested to increase understanding of muscle function and muscle disease and to ensure an efficient bench-to-bedside transition of promising technology.   

In her role, Marita is close to affected individuals and their families with a range of muscular dystrophies and related neuromuscular conditions and understands their “sense of urgency” to find treatments. She believes that research is now entering an exciting phase and that it is vital that patient organisations work together with the scientific community to have an input in the prioritisation and development of therapeutic approaches.

Marita has also worked as a scientist within molecular genetics for more than 15 years and was involved in developing a gene therapy approach for Duchenne muscular dystrophy. She can appropriately communicate the advances and progress to patients and their families.

 

Elizabeth Vroom



United Parent Projects Muscular Dystrophy (UPPMD) is owned and managed by parent project organisations set up by parents of children with Duchenne Muscular Dystrophy in many different countries all over the world. These national projects are run by parents for parents. UPPMD is managed by parents who lead the national parent projects.

The organisation came together after we realised that there was no sense in working in isolation from each other. We all share the same aims and dreams and they can be realised more efficiently using collective experiences and resources.
 

Elizabeth Vroom is the president of the Dutch Duchenne Parent Project and the chair of the worldwide United Parent Projects Muscular Dystrophy (UPPMD). She is a member of the TREAT-NMD Project Ethics Council as well as the TACT committee and is a practising Orthodontist.

Elizabeth is invited to speak at many international conferences about DMD and related issues.

 
 




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