Conference reports

ZNM- Zusammen Stark! e. V. also funds the participation of its members  in conferences on centronuclear myopathies.

The reports of the attending members are published here.

27 Nov – 29 Nov 2017 conference Treat-NMD in Freiburg, Germany

Representing ZNM-Zusammen Stark! e. V.  Arlene Wüstner and Aart den Hollander  attended the conference of Treat-NMD in Freiburg.

Treat-NMD was founded 10 years ago, to improve the co-operation of researchers, pharmaceutical companies and patient advocates and to speed up the development for rare neuromuscular disorders.

This has been successfully achieved in parts. As of today there are two approvals for therapies for neuromuscular disorders: one for Duchenne and another one for SMA. These treatments help a part of the patients with those disorders. Now is the time to extend the knowledge of these treatments to help even more children.

In addition, there is a clinical study running for a treatment for myotubular myopathy. Ana Buj-Bello gave a talk on this topic and everybody was very silent.

Ana Buj-Bello beim Vortrag

We learned also how a patient organization can register at the EMA (European Medicine Agency). When a treatment is approved in the E.U., the EMA gets into contact with ZNM – Zusammen Stark! e. V. . Patient advocates for Duchenne and SMA told us, that the EMA then will ask questions like: “When would you as patient say a treatment or a drug is successful?” Now, we as memners of ZNM- Zusammen Stark! e. V. can think about this question. If you have more questions please do not hesitate to contact us

Kind regards

Arlene Wüstner and Aart den Hollander

 

 

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21 – 23 July 2017 MTM-CNM conference in Nashville, USA

Every two years the MTM-CNM family connection organizes a family conference on centronuclear myopathies in the USA. Representing our association, ZNM – Zusammen Stark! e. V., Arlene Wüstner went to Nashville. In this way, she could get into contact with the US-amarican families and learn about recent developments in research on therapies for CNM.

She writes herself: “Viel zu schnell war die 5. MTM/CNM Family Konferenz der MTM/CNM Family Connection wieder zu Ende. Auf der zweijährig stattfindenden Familien-Konferenz mit über 200 Teilnehmern trafen sich alte Freunde und fanden sich neue. Es gab viel Zeit für den persönlichen Erfahrungsaustausch und ein neuer MTM-Warrior und seine Familie aus Belgien wurden herzlichst in die Gemeinschaft aufgenommen. Natürlich standen auch in diesem Jahr Experten und Wissenschaftler Rede und Antwort zu ihren aktuellen Projekten und bisherigen Forschungsergebnissen. Eine Vorstellung der Wissenschaftler und der teilnehmenden Familien findet ihr im Programmheft.

Zmore on the conference you can find here: http://www.mtm-cnm.org/2017.html

The pdf with the program you will find here : http://www.mtm-cnm.org/uploads/3/7/6/9/37691413/2017_mtm-cnm_family_conference_web.pdf