NOVARTIS
18.6.2020 01:03:14 CEST | Business Wire | Press release
STRICTLY FOR EUROPEAN MEDICAL AND PHARMACEUTICAL TRADE MEDIA ONLY
Many patients with SCD only live into their 40s and endure unpredictable pain crises which disrupt their lives physically, socially and emotionally.1 It is a genetic condition which is life-long and rising across Europe.2 Despite the World Health Organization recognising SCD as a global problem, knowledge about SCD among healthcare providers and the public is poor.3 SCD remains an invisible health issue in Europe and Novartis is dedicated to help make SCD a healthcare priority in Europe.
“SCD is a neglected chronic disease of increasing global health importance. There is a lot of work to be done to help the growing numbers of patients in Europe,” Professor Béatrice Gulbis, Co-coordinator of the European Reference Network EUROBLOODNET and specialist of inherited rare anemias. “It is important for healthcare professionals, policymakers, patients and the public-at-large to understand the severity of sickle cell disease and take a European-wide collaborative action on education, funding for research and awareness, and guidance and tools for screening and follow-up.”
SCD is a genetic blood disorders affecting millions of people worldwide. 4 It is a life-long and debilitating disease.5 It also has a substantial impact on patients’ emotional well-being and daily life affecting their ability to work and complete education.6 SCD financially affects the individual and their family; society and healthcare systems. 7 The burden of blood disorders in Europe is €23 billion per year, a level of cost that is not matched in current European haematology research funding.7
Early diagnosis and regular medical care can prevent complications and contribute to improved life expectancy and quality of life, however, a recent survey revealed that many patients don’t seek care despite symptoms and complications due to previous poor experience at hospital (39%) with some reporting that healthcare providers do not understand the disease (26%).6,8 Novartis is calling for a European-wide collaborative action on education, funding for research and awareness; and make sure that the patients receive equal and homogeneous healthcare access and treatment across Europe .
Tomorrow, a panel of EU experts and patients will be coming together for a one-hour webinar to highlight the lack of awareness and mis-conceptions of SCD amongst policy makers and the public in Europe. The meeting aims to highlight the action required to help improve the lives of those living with this debilitating disease. The experts will explore:
- SCD and the complexity of its diagnosis and management
- The burden of SCD on quality of life for patients and caregivers
- The impact of the COVID-19 pandemic on existing and emerging rare diseases in Europe
- Why there is a need for urgent strategy to address equal access to treatment in Europe
The expert panel comprises:
- Miriam Santos Freire, Patient Advocate
- Mariane De Montalembert, Healthcare Professional – (Eurobloodnet, ERN)
- Simone Boselli, Public Affairs Director – (European Organisation for Rare Diseases, EURORDIS)
- Yolande Adjibi, Head of Patient Relations in Sickle Cell Disease – (Novartis, Region Europe)
Yolande Adjibi, Head of Patient Relations in Sickle Cell Disease at Novartis comments: “For over 40 years, Novartis has been committed to understanding SCD and working towards treatment. We aim to support patients, healthcare providers and caregivers in their experiences with this debilitating disease. However, more needs to be done. We need to start conversations which will spark a positive change for SCD patients.”
In 2016 The European Hematology Association identified a need to improve treatment strategies for both acute and chronic complications of SCD to optimise patient care, in order to positively affect patient health and quality of life, and to reduce hospitalisation length, patient disability, and cost of care but limited progress has been made.7 As COVID-19 poses an additional threat to the lives of SCD patients and subsequently healthcare systems across Europe, urgent action is required to help improve the lives of those affected by SCD and to guide healthcare systems towards early diagnosis and equal access to treatment.
To join the conversation and pose questions directly to the panel please register here: https://us02web.zoom.us/webinar/register/WN_NB8ARtG2SMqJiCcM8MC9Kg
- END –
About Sickle Cell Disease
Sickle cell disease is a debilitating inherited genetic blood disorder that affects the shape of the red blood cells and can make blood cells and blood vessels stickier than usual.9 ,10 When blood cells stick to one another, they can form multicellular adhesion clusters in the bloodstream. These clusters can reduce and block the flow of blood and oxygen, which can cause damage to the blood vessels and lead to acute and chronic complications.6,9 These blockages also can lead to painful crises called VOCs, which are considered the clinical hallmark of the disease and the main reason why patients seek medical care in hospitals.9
About Novartis
Novartis is reimagining medicine to improve and extend people's lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world's top companies investing in research and development. Novartis products reach more than 750 million people globally and we are finding innovative ways to expand access to our latest treatments. About 105 000 people of more than 140 nationalities work at Novartis around the world. Find out more at www.novartis.com .
References
1 Adegbola M, et al. 2012. Voices of adults living with sickle cell disease pain. J Natl Black Nurses Assoc. 23(2), pp.16-23.
2 Roberts I, de Montalambert M. 2007. Sickle Cell disease as a paradigm of immigration haematology: new challenges for hematologists in Europe. Haematologica 92(7)pp.865-71.
3 Mburu, Joy; Odame, I. 2019. Sickle Cell Disease: Reducing the Global Disease Burden. Int J Lab Hematol (41) 1:82-88.
4 Jain D, Lothe A, Roshan C. 2015. Sickle cell disease: current challenges. Journal of Hematology & Thromboembolic Diseases . 10. Doi: 10.4172/2329-8790.1000224.
5 Steinberg M. 1999. Management of sickle cell disease. N Engl J Med . 340(13):1021-1030.
6 Osunkwo I, Andemariam B, Inusa B, et al. Management Strategies and Satisfaction Levels in Patients With Sickle Cell Disease: Interim Results From the International Sickle Cell World Assessment Survey (SWAY). Poster presented at: The American Society of Hematology Annual Meeting; December 7-10, 2019; Orlando, FL.
7 European Hematology Association (EHA) Roadmap for European Hematology Research: A Consensus Document (2016), Available at: http://www.haematologica.org/content/101/2/115 (Accessed June 2020).
8 National Heart, Lung and Blood Institute. Sickle Cell Disease. Available at: https://www.nhlbi.nih.gov/health-topics/sickle-cell-disease (Accessed June 2020).
9 Gutsaeva D, Parkerson J, Yerigenahally S, et al. 2011. Inhibition of cell adhesion by anti-P-selectin aptamer: a new potential therapeutic agent for Sickle Cell Disease. 13;17(2), pp.727-35.
10 Sparkenbaugh E, Pawlinski R. 2013. Interplay between coagulation and vascular inflammation in sickle cell disease. Br J Haematol. 162(1). Pp.2-14.
View source version on businesswire.com: https://www.businesswire.com/news/home/20200617005698/en/
Link:
About Business Wire
Subscribe to releases from Business Wire
Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.
Latest releases from Business Wire
Andersen Consulting tilføjer samarbejdsfirmaet TechSparq25.10.2026 06:09:00 CET | Pressemeddelelse
Andersen Consulting udvider sine kompetencer inden for teknologi og forretningstransformation gennem en samarbejdsaftale med TechSparq, en amerikansk virksomhed, der kombinerer strategi, commerce engineering og ai med praktisk implementering. TechSparq er et digitalt konsulenthus, der tilbyder strategi- og rådgivningstjenester, commerce engineering samt tjenester inden for ai og agentbaseret handel til virksomheder inden for detailhandel og forbrugsgoder, luksus og mode samt e-handel og direkte salg til forbrugere. Virksomheden har hovedkontor i Oregon og kombinerer strategisk planlægning med praktisk implementering for at hjælpe kunderne med at udvikle deres digitale initiativer, kundeoplevelse og driftsmodeller. "Vi etablerede vores virksomhed for at bygge bro mellem dem, der udvikler strategien, og dem, der har ansvaret for at føre den ud i livet," udtalte Dedrick Boyd, stifter af og administrerende direktør for TechSparq. "Vores samarbejde med Andersen Consulting giver os mulighed
BeOne Presents Compelling Data in Hard-to-Treat Liver and Lung Cancers at ESMO 202624.10.2026 10:15:00 CEST | Press release
Potential first-in-class GPC3 x 4-1BB bispecific achieves a 36% confirmed response rate at Phase 3 dose and favorable safety profile in heavily pretreated liver cancerBrain-penetrant PRMT5 inhibitor shows promising antitumor and CNS activity in the largest publicly shared MTAP-deficient datasetFirst clinical data of CEA-targeting ADC demonstrates encouraging and sustained responses in CEAhigh NSCLC BeOne Medicines Ltd. (Nasdaq: ONC; HKEX: 06160; SSE: 688235), a global oncology company, today announced new data from multiple differentiated investigational medicines across its priority solid tumor disease areas at the European Society for Medical Oncology (ESMO) Congress 2026 (October 23–27, Madrid). The presentations span gastrointestinal, lung, and breast cancers, and reflect the growing momentum of BeOne’s solid tumor franchise. Mark Lanasa, M.D., Ph.D., Chief Medical Officer, Solid Tumors at BeOne, said: “At ESMO, we are showing how our strategy of matching the right modality to dise
Phase 3 RAINIER Results Supporting Best-in-Class Potential of Povetacicept in IgA Nephropathy Published in the New England Journal of Medicine and Presented at the American Society of Nephrology Kidney Week 202623.10.2026 19:42:00 CEST | Press release
- Vertex is on track for the U.S. FDA Prescription Drug User Fee Act (PDUFA) date of November 30 for potential accelerated approval of povetacicept –- Data published in NEJM and presented at ASN highlight povetacicept’s unique trifecta of efficacy, favorable safety, and the simplicity of dosing at-home every 4 weeks with a low-volume autoinjector –- Data presented at ASN include a qualitative study examining patient perspectives on current and emerging IgAN therapies, showing that 96% of participants preferred an autoinjector over a prefilled syringe, and 80% of participants preferred dosing every 4 weeks versus weekly dosing - - New clinical studies of povetacicept underway including a study evaluating patient experience on povetacicept administered via autoinjector - Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the publication of Phase 3 RAINIER results for povetacicept in the New England Journal of Medicine (NEJM), presentation of these data at the American Soc
BeOne Medicines Reports Sustained Survival Benefit with TEVIMBRA-ZIIHERA Combination in First-Line HER2+ Gastroesophageal Cancer23.10.2026 16:15:00 CEST | Press release
New data from HERIZON-GEA-01 Phase 3 trial show 36-month overall survival of nearly 40% with TEVIMBRA plus ZIIHERA and chemotherapy, and median OS reaching 27 monthsTEVIMBRA-containing arm was associated with a four-month longer median OS compared with ZIIHERA plus chemotherapy BeOne Medicines Ltd. (Nasdaq: ONC; HKEX: 06160; SSE: 688235), a global oncology company, today announced new survival results from the planned second interim analysis (IA2) and final formal analysis of the Phase 3 HERIZON-GEA-01 trial, further confirming the benefit of TEVIMBRA® (tislelizumab) plus ZIIHERA® (zanidatamab) and chemotherapy as first-line treatment for patients with HER2-positive locally advanced or metastatic gastroesophageal adenocarcinoma (GEA). The results will be presented today as a late-breaking abstract during a rapid oral session at the European Society for Medical Oncology (ESMO) Congress 2026 in Madrid, Spain (LBA79). Mark Lanasa, M.D., Ph.D., Chief Medical Officer, Solid Tumors at BeOne,
Lifezone Metals Confirms Advanced Negotiations with Orion CMC on Kabanga Nickel Project23.10.2026 16:10:00 CEST | Press release
Lifezone Metals Limited (NYSE: LZM) notes comments made today by Oskar Lewnowski, Founder and Group Chief Executive of Orion Resource Partners, in an interview with Bloomberg Television regarding the Company’s Kabanga Nickel Project in Tanzania. In response to inbound inquiries from media outlets and investors following the interview, the Company can confirm that negotiations with Orion Critical Mineral Consortium (Orion CMC) regarding a significant equity investment into the Kabanga Nickel Project are at an advanced stage. The Orion CMC is a global investment platform launched by Orion Resource Partners in partnership with the U.S. International Development Finance Corporation (DFC) and Abu Dhabi's sovereign investor ADQ. As previously announced, the Company has been running a competitive process, led by Standard Chartered Bank, to secure a strategic investment into the Kabanga Nickel Project. Social Media LinkedIn | X | YouTube About Lifezone Metals Lifezone Metals (NYSE: LZM) is com
In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.
Visit our pressroom
