After the success of the stages of Bologna and Bassano del Grappa (Vicenza), arrived in Bari on 13 September ‘Rare Nights’, the information initiative on paroxysmal nocturnal hemoglobinuria (Epn) promoted by Sobi, a multinational biopharmaceutical company, leader in the development of innovative therapies aimed at improving the lives of people with rare diseases. Rare Nights – the company recalls in a note – is a music initiative dedicated to the 350 Italians with Epn, which takes shape thanks to the notes played under the stars by Eleonora Montagnana, a young classical violinistwith over 300 thousand followers on TikTok, also known thanks to its participation in television programs, recordings and concerts alongside nationally and internationally renowned artists (WATCH THE VIDEO).
The musician and influencer gave life to a night performance – as a reminder of the EPN’s ‘nocturnal’ feature – starting at 10pm in one of the most evocative and scenic areas of Bari: Piazza del Ferrarese, the main access point to the ancient village and a place of great charm. The live performance, on the notes of ‘Sweet Child O’ Mine ‘and other songs, was accompanied by a rich social programming on the artist’s channels – TikTok, Instagram and Facebook – to give users the opportunity to enjoy the performance also from home. “I am happy and proud to be able to take part in an initiative such as Rare Nights – says Montagnana – I hope my music can be useful not only for a social awareness of this ultra rare disease, but also to give a little lightness and a sweet awakening to people with Epn “.
Paroxysmal nocturnal hemoglobinuria is a disease characterized by episodic nocturnal and morning manifestations, with symptoms that are not always evident: to recognize them you need to pay attention to your body, literally ‘listening’ as in the case of a melody, a symphony of notes. The goal of Rare Nights is to shed light on this ultra-rare chronic disease affecting between 0.5 and 2 people per million inhabitants. Epn – characterized by anemia (low hemoglobin), an increased risk of thrombosis, and debilitating symptoms – occurs when the cells in the bone marrow responsible for producing red blood cells (which help carry oxygen around the body) mutate and produce blood cells. defective blood. The debilitating symptoms of this blood disorder include, among others, fatigue, a sense of profound exhaustion that affects 96% of patients, difficulty concentrating, abdominal pain, dyspnea. Recognizing and diagnosing the PN quickly is important to define an effective therapeutic path and guarantee a concrete improvement in the quality of life of these people, who perform even the simplest daily gestures with great difficulty.
“Paroxysmal nocturnal hemoglobinuria, if not recognized and untreated, can produce serious consequences, even permanent – he explains Alessandra Ricco, medical director, Uoc Hematology with transplant, Policlinico di Bari – For this reason it is important to keep alive the interest in this condition not only in the professionals, as its prompt recognition and treatment protects the health and the very life of the people who are affected by it. Just as music is transmission and reception in a contemporary and continuous flow, so the relationship between people linked – each in their own role – from the world of rare disease is communication and listening, of symptoms, life experiences, needs “.
As the specialist points out, “it is the task of research to translate listening into understanding phenomena and, therefore, into actions aimed at restoring the natural balance that the disease has broken. Ever since EPN has become a treatable disease – highlights Rich – the quality and life expectancy of those affected has radically changed; research has then produced drugs that can be administered at longer intervals of time. The new challenges concern the production of molecules with diversified targets and routes of administration, which are increasingly specific, effective and designed on the needs of the individual subjects involved “.
For people with Epn they exist supportive therapies, i.e. treatments that do not allow healing but lessen symptoms, reduce complications and improve quality of life. These include C5 complement inhibitors (C5i), folic acid, iron and vitamins, prophylaxis or anticoagulant therapy, erythropoietin, transfusions. Approved in 2021 by the European Medicines Agency Ema and in August 2022 by the Italian Medicines Agency Aifa, pegcetacoplan (Aspaveli *) is also available, the first inhibitor of C3 (C3i), a new class of therapies that inhibit complement by affecting the C3 which is located upstream of C5 in the complement cascade. Inhibition of the C3 target allows to control, in addition to intravascular haemolysis, also the destruction of red blood cells in the spleen and liver (extra-vascular haemolysis).
“Life with a rare disease – he says Giampiero Marra, medical director Sobi Italy, Greece, Cyprus and Malta – it is often veiled by a shadow that derives from the symptoms of the disease itself: fatigue, anemia, brain fog can really compromise the quality of everyday life and prospects for the future. Sobi, who as always cares about people with rare diseases, wants to bring a touch of lightness and delicacy to this community. This is why we have chosen a different communicative register. Thanks to the notes of a violin, Rare Nights will invite the public and people with Epn to literally ‘listen’: the first in listening to others, as a sign of openness to understand the difficulties and the often invisible weight of this pathology; the latter listening to their own body, to realize how much quality of life they can still achieve thanks to therapeutic innovation “.
The Bari stage is the last of three evenings scheduled from June to September: the first was held in Bologna on Tuesday 28 June, the second in Bassano del Grappa on 12 July.