0-TESTO COMPLETO.pdf - Fondazione Santa Lucia

the intrinsic condition of disease rarity. Don Gnocchi Foundation has 29 rehabilitation
centers equally distributed in many Italian regions, hence it may
provide a unique opportunity to standardize rehabilitation in rare diseases.
1. Pareyson et al. (2006) Neuromolecular Med 8:3-22.
2. Pareyson et al. (2006) Pharmacol Res 54:436-441.
3. Lindeman et al. (1995) Arch Phys Med Rehabil 76:612-620.
What the project adds to the information already available
The project will provide:
1) information on the current rehabilitation access for patients affected
by a rare disease, like CMT, in the different Italian regions;
2) standardization of rehabilitation and efficacy evaluation according to
the best available approach based on scientific knowledge.
The main tool will be the creation of a rehabilitation network able to provide
a forum for collaboration, communication and consensus-building that
enables the providers of rehabilitation services to achieve an integrated rehabilitation
system and coordinate service, promote equitable access, address
gaps, increase research and education, and measure overall performance.
One of the key tools of the network will be a website.
OBJECTIVES
RF07.41 – Access equity and qualitative standard of rehabilitation in rare diseases…
The main objectives are:
1. To evaluate the current access of CMT patients to rehabilitation therapy.
2. To reach a consensus on the best rehabilitation approach for CMT
(definition of a core of standards).
3. To establish a network of centers (with team of physiatrists, physiotherapists
and neurologists with a selective expertise in CMT) that take care
and perform rehabilitative standardized treatment for CMT patients and
ensure that the standard is met, and continuously improved. To assess the
efficacy of the network in improving access of CMT patients to rehabilitation
therapies.
The secondary objectives are:
1. To develop and reach a consensus on the best outcome measures to
assess efficacy interventions (particularly as responsiveness is concerned).
2. To develop and apply, in a subgroup of CMT patients, a quantitative
and objective protocol for the evaluation of treatment efficacy by means of
Gait Analysis techniques.
3. To test the efficacy of one treatment and evaluation protocol, structured
as a pilot study.
4. To test the clinical variables that may influence rehabilitation or that
may be influenced by rehabilitation (as motor patterns and pain).
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