(Adnkronos) - L'ipoparatiroidismo è un raro disturbo endocrino, non abbastanza conosciuto, caratterizzato da deficit totale o parziale di secrezione di paratormone (Pth) responsabile dell’omeostasi dei livelli di calcio e fosforo nel sangue. Al media tutorial ‘Ipoparatiroidismo: inquadramento epidemiologico e clinico, nuove prospettive di trattamento e burden della patologia’, organizzato a Milano, oltre ad un inquadramento clinico ed epidemiologico, si è esplorato il burden della patologia per le persone affette da questa malattia.
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00:00Hypoparathyroidism is a rare disease with an incidence of about 0.8-2.3 new cases every 100,000 people per year, characterized by an inadequate secretion of parathormones.
00:17This pathology was discussed at the Media Tutorial organized in Milan by Ascendis Pharma.
00:23Hypoparathyroidism is a condition, as the word says, in which endocrine glands that are called parathyroids do not work.
00:31As we talk about hypothyroidism in the thyroid, for the parathyroids we talk about hypoparathyroidism.
00:37When the parathyroid glands, which are four small glands that are right behind the thyroid, for this reason they are called parathyroids, do not work, the parathyroid hormone is not secreted.
00:49It is also abbreviated with the name PTH, the patients know well this acronym PTH.
00:56And when the PTH is low or does not work on the receptor, we have a series of consequences, because the parathyroid is the main hormone that controls the calcium in the blood,
01:06it is what keeps our calcium normal, and calcium is a fundamental ion for life.
01:11Experts have delved into various aspects of the pathology, from epidemiology to the clinical framework, up to the complications and the most recent therapeutic prospects.
01:20It was a great innovation to have a drug that is the right drug for this pathology, which until now we have treated with surrogates, we have tamponed and treated the symptom.
01:32Now we have a therapy that replaces the hormone in an adequate way and that allows to cover the calcium for all 24 hours.
01:41It is a fragment of the PTH of the parathyroid hormone, which is linked to a vector, manages to work for 24 hours and therefore guarantees not only the adequate calcium for the patient,
01:52but also guarantees the management of what are the comorbidities that the patient can develop over time,
01:59because with the excess of calcium and calcitriol that we are using now, the patient can go to nephrocancinosis.
02:05The Milan Media Tutorial confirms the commitment of Ascendis Pharma to counteract rare diseases such as lipoparathyroidism, with particular attention to the needs of patients.
02:15Ascendis was born in 2007, focusing on the development of drugs for endocrinological rare diseases.
02:21Currently, the developed company has had the authorization of two drugs from the European Commission and in Italy we have had the privilege and the good fortune to have three centers involved,
02:31both in the study of phase 2 and in the study of phase 3, as regards the development of the replacement therapy for lipoparathyroidism.
02:37So we are committed and we want to ensure that patients in Italy have access to a therapy that could revolutionize their lives.