(Adnkronos) - “Da circa 15 anni abbiamo iniziato lo sviluppo di asfotase alfa, una terapia enzimatica sostitutiva per il trattamento della ipofosfatasia. Ci siamo assunti dei rischi nell’ambito della ricerca per l’ipofosfatasia, ma ne siamo orgogliosi e oggi possiamo dire che ne è valsa la pena perché siamo riusciti a portare ai pazienti questa importante opzione terapeutica”.
Lo ha detto Federica Sottana, senior country Medical director di Alexion, AstraZeneca Rare Disease, partecipando, questa mattina a Milano, all’incontro con la stampa sulla 'Svolta nella cura dell'Ipofosfatasia: approvata la rimborsabilita' in Italia di asfotase alfa, la prima terapia per il trattamento di bambini e adulti', organizzato dalla faramceutica.
Lo ha detto Federica Sottana, senior country Medical director di Alexion, AstraZeneca Rare Disease, partecipando, questa mattina a Milano, all’incontro con la stampa sulla 'Svolta nella cura dell'Ipofosfatasia: approvata la rimborsabilita' in Italia di asfotase alfa, la prima terapia per il trattamento di bambini e adulti', organizzato dalla faramceutica.
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00:00For about 30 years, Alexion has been committed to research to develop innovative and transformative therapies for people affected by rare diseases.
00:17For about 15 years, we have started the development of a substitutional enzymatic therapy for the treatment of alpha-asphotase,
00:27a rare genetic disease due to a genetic alteration that determines an enzymatic deficit and the missing enzyme is alkaline phosphatase,
00:42which is a fundamental enzyme for mineralization and bone growth.
00:47Alexion does not stop at the treatment, it tries to assist and support patients, patient associations and the scientific community throughout the course of the disease,
01:00from diagnosis to what is called beyond the pill, so services beyond the drug.
01:06For a few months now, Alexion has proposed to the scientific community a completely free service called HPP Easy Program,
01:19which is a service that provides a specialized, certified laboratory that performs tests to dose alkaline phosphatase,
01:34B6 vitamin substrates such as IPA and inorganic pyrophosphate.
01:43In addition to this project, which facilitates and speeds up a premature and tempestuous diagnosis of the pathology,
01:52Alexion supports a service called ARCO, which provides the drug at the patient's home,
02:02provides virtually nurses who guide the patient in the self-supply of the drug, but also guide and monitor the patient in the process of adherence to the treatment.
02:18In addition to this, psychological support, support from a nutritionist and possibly a physiotherapist is provided.