The MYOCURE project website is now available

New therapies for rare diseases: New EU project MYOCURE advances treatment of rare hereditary muscle disorders.

New therapies for rare diseases: New EU project MYOCURE advances treatment of rare hereditary muscle disorders.
May 30, 2016

The new collaborative
research project MYOCURE aims at advancing the development of new therapies for
patients with rare muscle diseases. In particular, MYOCURE will impact on an
estimated 20,000 people in the EU suffering from myotubular myopathy (MTM) and
glycogen storage disorder (GSD) type II (Pompe’s disease) as well as impact on
other muscle disorders. MTM and GSD II are quintessential hereditary muscle diseases
for which there is no definitive cure. They are attractive diseases for gene
therapy as they comprise a diverse family of rare genetic diseases typically
caused by single gene defects, and often provoke significant morbidity and
mortality due to skeletal muscle, cardiac and/or diaphragm dysfunction. The
costs for treatment and care taking of GSD II patients, amount to €400,000 to
€700,000 per year per patient. In the EU, this corresponds to an estimated cost
of €4 billion to €7 billion annually.
Thus, advancing research in this field is a societal need and a crucial
commitment to improve patient care and relieve the healthcare-system.

In this context,
MYOCURE was launched to overcome current scientific and societal bottlenecks,
as well as to develop an
innovative and
clinically applicable gene therapy platform to cure rare hereditary muscle
disorders. Equipped with a budget of about €6 million, the novel approaches
that will be developed in MYOCURE shall form the basis for a one-time treatment, which is expected to
provide enhanced patient treatment and significantly reduce healthcare costs.

Professor Marinee
Chuah from the Vrije Universiteit
and Coordinator of the project stresses the importance of the
social and economic impact of the
project: “Establishing such a clinically
translatable one-time treatment platform will contribute significantly to a
higher patients” quality of life. Moreover, the social impact stretches out to
the families and relatives of the affected persons, as the need for home or
hospital care is decreased, hence reducing the overall social and economic
She further emphasizes that one of the unique strengths of MYOCURE is the
combination of most recent innovations in gene therapy with the latest insights
in immune control to maximize efficacy and safety. Furthermore, MYOCURE
will enhance expertise in the field of orphan and rare diseases, as well as strengthen
the competitiveness and growth of companies in the EU by developing innovations
meeting the needs of European and global markets.


We participate in MYOCURE project as a regulatory partner leading the following

– Scientific Advice with the EMA for
the treatment of MTM and GSD II.

– Orphan Drug Designation Applications
for both indications, MTM and GSD II.

– Consolidation of a clinical
development plan to be agreed with the EMA CHMP in a Follow-Up Scientific

brings together leading experts in science and industry from eight European
partner institutions to reach these goals and successfully implement the
project’s ambitious work plan. 

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We arean International Scientific and Regulatory Affairs consultancy, with offices in Barcelona and Munich.

We offer comprehensive services for Drug Development and Regulatory Affairs to Pharma, Biotech and Medical Devices companies.

Since 2000, we have consistently grown and now have over 70 professional consultants with backgrounds in Life sciences (Pharmacy, Chemistry, Biology, Biochemistry, Biotechnology, Medicine and Veterinary Medicine).

Our consultants are experts in their field and are in direct contact with EU agencies (EMA and NCAs) for the development and implementation of new regulatory standards.


MYOCURE: This project has received funding from the
European Union?s Horizon 2020 research and innovation programme under grant
agreement No

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