“The clinical approach to rare diseases is a holistic, complex approach that must consider a series of aspects. Rare disease patients have a series of comorbidities, associated conditions. For example, a patient with a neurological disease or with a rare epilepsy, especially in childhood, also has a cognitive and motor disability. Therefore, the clinical approach must be multidisciplinary: multiple health specialists around the individual patient and family can offer an efficient and quality service that tends to improve the patient's quality of life. The next aspect is related to diagnostics, how to improve the quality of life and the therapeutic approach. Early diagnosis is essential as it allows for personalized treatment.” This was stated by Nicola Specchio, head of the Uoc Neurology of epilepsy and movement disorders and director of the research unit in neurological and neurosurgical diseases of the Bambino Gesù pediatric hospital, on the occasion of the Adnkronos Q&A event 'Health and healthcare, a shared challenge', today in Rome.
“Another fundamental point to consider when talking about multidisciplinarity and when talking about the approach to rare neurological diseases is the one defined by the Pdta – adds the neurologist – a fundamental tool that we must use quickly for various diseases”. A separate chapter, clinical research and experimental therapy. “To obtain experimental treatments we need to streamline the procedures for starting clinical trials. We need support from regulatory bodies to provide patients with the most modern and innovative therapeutic options”, he concludes.
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