Swedish digital startup company Pilloxa today announced that it is to collaborate with the Nordic office of Chiesi Global Rare Diseases on using the Pilloxa platform to support the treatment journey for patients with nephropathic cystinosis across Scandinavia.
Nephropathic cystinosis is an ultra-rare, multisystemic, genetic disorder characterized by the accumulation of the amino acid cystine in the lysosomes within the cells. This accumulation leads to damage in various tissues and organs of the body including kidneys, eyes, muscles, liver, pancreas and brain (1). The continuous progression of the disease, together with the high medication burden, places large demands on the patients and their caregivers. Most younger patients, under 11 years of age, follow the prescription correctly. (2) However, as with other chronic diseases, many teenagers, and young adults with nephropathic cystinosis are struggling to follow the treatment regimen. Strategies such as better information on the disease, patient self-care promotion, and facilitated transition to adult healthcare services have been suggested to improve compliance and the clinical management of cystinosis. (2)
To learn more about nephropathic cystinosis, please see https://rarediseases.org/rare-diseases/cystinosis/
With the app Cool 2B Rare_C we are aiming at helping patients to take control of their disease and treatment. In the app, patients and caregivers, such as family members, will be able to:
- set reminders on when to take the medication
- follow the adherence over time
- have easy access to information about the disease and related topics.
“We are honored to work with the Nordic office of Chiesi Global Rare Diseases to support patients, an incredible partner within rare diseases aiming to improve the lives of people across the world. Digital solutions have a great potential to give personal support to chronically ill patients at every step on the treatment journey, to give hope and motivate them in their everyday battle. Together we are at the forefront of a new paradigm within personalized healthcare, and we are just getting started. We strongly believe that this will increase the quality of life for patients with nephropathic cystinosis and hope to ease the transition of care into adult life for many patients,” commented Helena Rönnqvist, CSO Pilloxa.
“Our approach is to develop and test solutions that we believe will benefit patients, caregivers and health care professionals in diagnosing patients early as well as managing a rare or ultra-rare disease. We look forward to receiving feedback on this app from the nephropathic cystinosis community.” Patrick Svarvar, Head of Rare Diseases, Nordics Chiesi Global Rare Diseases
“Patients are the beginning and the end of our journey. In the work developing this app, Pilloxa has interviewed patients, caregivers and physicians to get an insight on the gaps that need to be filled in order to improve the treatment journey. We would like to thank all of them for giving their time as well as sharing their knowledge on how it is to live with nephropathic cystinosis.” Brita Forsberg, Medical Advisor, Nordics Chiesi Global Rare Diseases
About Chiesi Global Rare Diseases
Chiesi Global Rare Diseases is a business unit of the Chiesi Group established in February 2020 and focused on research and development of treatments for rare and ultra-rare disorders. The Global Rare Diseases unit works in collaboration with Chiesi Group to harness the full resources and capabilities of our global network to bring innovative new treatment options to people living with rare diseases, many of whom have limited or no treatments available. The unit is also a dedicated partner with global leaders in patient advocacy, research and patient care. For more information visit https://www.chiesiglobalrarediseases.com/
About Chiesi Group
Based in Parma, Italy, Chiesi is an international research-focused pharmaceuticals and healthcare group with over 85 years’ experience, operating in 30 countries with more than 6,000 employees (Chiesi Group). To achieve its mission of improving people’s quality of life by acting responsibly towards society and the environment, the Group researches, develops and markets innovative drugs in its three therapeutic areas: AIR (products and services that promote respiration, from new-born to adult populations), RARE (treatment for patients with rare and ultra-rare diseases) and CARE (products and services that support special care and consumer-facing self-care). The Group’s Research and Development centre is based in Parma and works alongside 6 other important research and development centres in France, the U.S., Canada, China, the UK, and Sweden to promote its pre-clinical, clinical, and regulatory programmes. Chiesi, since 2019, is the world’s largest B Corp certified pharmaceutical group. The global B Corp movement promotes business as a force for good. The Group is committed to becoming carbon neutral by the end of 2035.
Chiesi was established in the Nordic region in 2014 and has operations in Sweden, Denmark, Norway and Finland.
To learn more about Chiesi Group’s Nordic subsidiary, visit https://www.chiesipharma.se/en/
The founders started Pilloxa after their Clinical Innovation Fellowship program in May 2014. The confusion and worry in patients following their treatments sparked their passion and they saw that their combined background in technology, medicine, and entrepreneurship could bring high impact. Stockholm proved to be a unique hub due to its presence of world leading healthcare organizations and access to top global talent. From their first steps with the Karolinska University Hospital to current partnerships with sector leaders, Pilloxa is now a European sector leader rooted in the Scandinavian peninsula.
To learn more about Pilloxa’s new product offering, explore their solutions at www.pilloxa.com
- Elmonem et al. Orphanet Journal of Rare Diseases (2016) 11:47
- Ariceta et al. Nephrol Dial Transplant (2015) 30: 475–480
Press Questions? Please contact Alexa Edstrom at or +46 76 567 3713 (Sweden)