Solutions for the management of severe neuromuscular diseases through

partnership between patient organization and professionals

EURORDIS 2019, BucharestIsabela Tudorache, Parent Project Association Romania

We are….

• A parent community based on common interests and aims, meant to support children and actively stepping into the course of the disease , founded in 2004

• A voluntary organization ( no payed personnel)

• We promote dialogue with all interested parts: medical, educational and governmental institutions, research and assistance institutions, private and non-governmental organizations which can contribute to a significant change

The challenge

• 650-700 patients with severe DMD/DMB / population 22 mil. - NO

center of expertize or a special NMD care center

• Passive attitude motivated by no cure available and the early death

• Many premature, early deaths – 16-20 years old

• A huge need of clear and complete route of medical services –

Standards of care

• no experience & poor financial resources for our small parent

association,

What we did:

Contact and learn from the fellow people worldwide, in

the same situation: Parent Project MD

Pat Furlong(USA) Filippo Buccela (Italy)

What we did: Partnership with medical professionals

• Dr. Amelia Dobrescu, Dr. Amelia Dobrescu, MD, PhD, Université de Médecine et de Pharmacie de Craiova,Centre Régional de Génétique Médicale

- our medical representative since 2005

What we did: Identify the relevant projects & networks of

excellence and LEARN

Funding from the European Commission and public-private partnerships .

We started our own Conferences…and descover there are hundreds like us

To enhance quality of life for patients and their families/caregivers, Parent Project

Romania established a partnership with several medical stakeholders:

Medical University Craiova & Carol Davila Bucharest, Hospitals Alexandru Obregia and

Victor Gomoiu from Bucharest, Hospital nr. 1 Craiova, Romanian society of Pediatric

Neurology, Society of Medical Genetics, the ro. Society of Pneumology and Somnology

Our initiative

1. Inform patients and their families about neuromuscular disease and its daily consequences : publish a Family Guide , Emergency Card, SoC materials,..

2. DMD National Patient Registry with online registration3. Act as interface between the family and the many specialists involved in

treating these pathologies: physicians, physiotherapists, social sector professionals.

4. Train/ Inform professionals to be aware of the specifics of neuromuscular diseases.

5. Identify the family's needs and help them to find solutions

2012-2014 the PP Service for multidisciplinary care in DMD/DMB, an experimental

project

,,Neuromuscular diseases: professional parents and patients”

UNIVERSITY OF CRAIOVA, ROMANIA

ASOCIATIA PARENT PROJECT, ROMANIA

FONDAZIONE OSPEDALI RIUNITI ANCONA ONLUS, ITALY

UNIVERSITY EDUCATIONAL CIRCLE, LATVIA

ACTION DUCHENNE LIMITED, UNITED KINGDOM

2-year Lifelong learning Grundtvig project for children, their families, adults with neuromuscular disorder.

Grundtvig Learning Partnerships2011-1-RO1-GRU06-14989 2

➢ Identifying, sharing and sharing good practices

➢ Creating educational materials: Guide for people

with neuro-muscular disorders, parents and

professionals.

➢Introducing the concepts of "family-centered care",

"multidisciplinary team care" and "parent-parent

educational approach"

Selected 5 specialists ,

Organize 1 week shadowing in specialized centers: Nemo Center

Italy and Stokolm, Karolinska Institute

Share experience &Practice a multidisciplinary care in Craiova

Our initiative: Train the multidisciplinary team !

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Do children with DMD transDo children with DMD

transition to adult health care services ?

2018 - Research ProjectWhat about respiratory care and management in Romania ?

From medical and patient perspectivewith Vanessa Ferreira(Por) & medical partners

ADVOCACY

Parent information sessions and support groups

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Key take away:

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CONCLUSIONS

management of severe neuromuscular diseases through partnership

Promote new practical models and advocate for change

3. Spread hope and acting models

2. Learn about the international practice and

SOC

Key learnings

• It is possible and feazable to organize multidisciplinary care for our children, and to stimulate public

services to implement it, but we also need a national standardization and recognition for neuromuscular

centres or hospital sections.

• To build their knowledge of modern standard of care, our doctors needs (training and) shadowing sessions

in more experienced Reference Centres.

• The international networks are essential to keep us updated with valuable and relevant information.

• The patient driven projects have more practical impact and highly motivated

• We need a better communication between eastern EU countries, not only in order to improve the research

for a cure, but also to improve our legislation inhealth and social servicies, pricing for defferent supportive

devices, care, and quality of our children & youngs .