Home-based technology for rare disease patients- Aparito’s approach for Late-Onset GM2 gangliosidosis

Late-Onset GM2 gangliosidosis (LOTS) is a rare and progressive neurological genetic disorder that has a devastating impact on those it affects. There is no cure and treatment for the disease currently focuses on managing symptoms.

Digital remote patient monitoring technology has been identified as having the potential to aid the drug development process. By developing new digital endpoints for clinical trials it will be possible to deliver healthcare and treatments tailored to the specific needs of patients. Innovative digital approaches for rare diseases can provide great opportunities for drug development, but they need to be suitable for the patients that will use them to ensure that sufficient data sets are generated.

In this white paper we summarise this innovative approach for a rare lysosomal storage disease (LSD) to show:

  • The potential application of remote patient monitoring technology for an LSD in adults.
  • The accessibility of the Aparito Atom5™ platform for patients with neurodegenerative diseases such as LOTS.
  • The importance of collecting real-world evidence for a rare disease to help develop a greater knowledge of the disease and its impact on the individual.
  • That LOTS patients have many unmet clinical needs that need to be addressed with emerging therapies.

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biomedical engineer with 5+ years experience in cancer clinical research. I am interested in using mHealth technologies to remotely monitor biological rhythms and symptoms in patients outside the hospital setting. I have developed expertise in large database management, biological time series collection and analysis from wearables. I have been involved in clinical trials support, design, conduct, monitoring and staff training.

New Data Scientist joins Aparito

Aparito welcomes Data Scientist Sandra Komarzynski to the team. Sandra joined in April 2020 and brings over five years’ experience in a wide range of clinical research areas.

I am a biomedical engineer with 5+ years experience in cancer clinical research. I am interested in using mHealth technologies to remotely monitor biological rhythms and symptoms in patients outside the hospital setting. I have developed expertise in large database management, biological time-series collection and analysis from wearables. I have been involved in clinical trials support, design, conduct, monitoring and staff training.

In my current role as Data Scientist at Aparito, I build analytical tools for health data using wearable devices and the Atom5™ platform for rare disease and cancer patients. My tasks involve data collection monitoring, data cleaning and analysis using tailored algorithms. In my most recent project, I focused on the feasibility assessment of remote surveillance of oncological patients during the Covid-19 outbreak – this involved the collection of Covid-related symptoms and physiological data recorded by a wrist-worn wearable device and the Atom5™ Platform. For this project, I was responsible for the assessment of patients’ engagement and adherence, analysis of the time series and the descriptive statistics.

Working at the French National Institute for Health and Medical Research (INSERM), I took part in a National project involving ten industrial, academic and clinical partners, that developed the first mobile platform for the remote multi-dimensional monitoring of chronic diseases [1]. I was responsible for linking the technical developments of the platform with the targeted users, including patients and health professionals; this included, managing the logistics, training, recruiting participants, monitoring the data collection and performing data analysis throughout the pilot tests in cancer patients. In my research role at Warwick Medical School within the Cancer Chronotherapy Team, I was involved in a study aimed at capturing the intra- and inter-variabilities in circadian rhythms of healthy volunteers [2]. The study recruited 30 participants who monitored their circadian rhythms for 1 to 3 weeks using telecommunicating devices such as a chest sensor, a wrist actigraph, and a bodyweight scale.

My further research in healthy volunteers was aimed at predicting the core body temperature from non-invasive physiological measures and questionnaires. I participated in the design of the study and was responsible for the technical support, data monitoring, management and analysis. Forty subjects were recruited and monitored, their core body temperature was measured using an eHealth ingestible pill, physical activity and skin temperature were recorded by a chest sensor during the subject’s daily life for a duration of one week. The subjects self-collected saliva samples for hormone level determination. The prediction model developed, was able to predict core body temperature phase from sex, score from a chronotype questionnaire, and two tele-monitored circadian parameters [3].

I was also involved in a French study that aimed to identify circadian and sleep disruption due to shift work in 200 nurses [paper in preparation]. The nurses provided a general questionnaire including demographics, work and medical history, sleep habits and medication use. They completed a chronotype questionnaire, wore a telemonitoring chest sensor and completed a precise work and sleep diary for a week. I coordinated the circadian rhythms analyses.

In order to find actionable determinants of circadian and sleep disruption in cancer patients, I helped design a multi-centre (5 in UK) observational clinical study. We investigated potential determinants of disruption in a number of tele-monitored circadian parameters, hormones and questionnaires. Patients took part in the study for 1 week of remote monitoring [paper submitted]. I was involved in the study set-up, clinical staff training, and study monitoring as well as the data validation, management and analysis. I developed analytics tools and was responsible for the time series analysis. Outside of work, I enjoy travel and photography.


  1. Maurice M et al., Innovative Project for Domomedicine Deployment: The PiCADo Pilot Project. eTELEMED 2015.
  2. Komarzynski S et al., Relevance of a Mobile Internet Platform for Capturing Inter- and Intrasubject Variabilities in Circadian Coordination During Daily Routine: Pilot Study. J Med Internet Res 2018
  3. Komarzynski S et al., Predictability of individual circadian phase during daily routine for medical applications of circadian clocks. JCI Insight 2019.
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Data Scientist joins Aparito

Aparito welcomes Data Scientist Clare Matthews to the team. Clare joined in May 2020 and brings 10 years of experience in applied mathematics and data analysis.

Clare has worked as a mathematician, data scientist and developer in both academia and industry. At the National Physical Laboratory (NPL) she provided statistical data analysis and modelling for a wide range of projects, such as the calibration of micro-thrusters used for steering satellites, optimisation of smart electricity grids, and data fusion for a network of sensors.

At the National Physical Laboratory she worked on a large European project to develop the capability for the dynamic calibration of pressure sensors, necessary to accurately measure pressure changes in engines. She developed a computational fluid dynamics model to aid in the design of the shock tube used to perform sensor calibrations and worked on signal processing techniques to calculate the uncertainty associated with the calibration. Clare also led the impact work associated with the project.

As a developer she worked on the creation of a big data platform for a large academic publishing company, pulling together data from multiple sources and generating statistics for an online reference management service.
She has worked with a telematics company and national insurance provider to create a vehicle crash detection algorithm for use on mobile and telematics devices. The algorithm was developed using data from incidents resulting in insurance claims, and uses sensors on a driver’s phone, or the blackbox device fitted to their vehicle, to trigger a real-time alert in the event that a serious crash is detected.

Her research career began at University College London, where she built a fracture-mechanics based model to study observations in seismic activity prior to eruptions at volcanoes with a long repose time.

More recently, at Bangor University, she has worked in the area of real-time summarisation of videos, primarily for the application of summarising daily events captured via wearable devices. Clare’s work at Aparito continues in this field of utilising video to capture data for medical purposes. Her focus is now on automatically rating eating skills from videos of mealtimes, allowing patients to be observed and assessed in their home environment.

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Chief Technology Officer joins Aparito

Aparito welcomes Aleksa Vukotic to the team as their new Chief Technology Officer. Aleksa brings detailed knowledge of technology within the finance, lifestyle and health sectors. 

Aleksa leads the Aparito technology team and is responsible for the technology vision and design of the Atom5™ platform.
Aleksa and his technology team at Aparito have designed, architected and implemented a cloud-native patient monitoring platform, using open source technologies and focus on automation and iterative delivery.

While developing and communicating technology vision and product strategy to internal and external stakeholders, he also participates in team efforts by maintaining a hands-on role and oversight of critical platform components.

Under Aleksa’s leadership, the Aparito has developed and is executing an ambitious roadmap, which includes a number of new features and improvements. The core focus of the innovative work are patient portal and self onboarding capabilities added to the Atom5™ platform, as well as world-class data platform, that will serve as needs of research and patient care focused organisations trying to make sense of remote patient monitoring data, with rich data analytics, machine learning and AI features.

As an experienced technologist and expert in cloud-native microservices, architectures and API design, Aleksa has  a track record as technology leader, speaker and author within the scope of Open Source, DevOps, BigData and NoSQL technologies across finance, lifestyle and health sectors. 
Recently Aleksa has acted as CTO and technology adviser for a number of startups in the FinTech and Health/Lifestyle sectors in the North West. Before this, he held senior technology roles in multinational financial institutions in London.

During his career, Aleksa has advised a number of large companies on Open Source technology, continuous integration and digital transformation, within the telecoms, oil, gas and retail sectors.
Aleksa is a published author: Neo4j in Action, published by Manning; Pro Spring 2.5 and Apache Tomcat 7, both published by Apress. In addition to regularly speaking at technology conferences and meetups, he is an active member of the UK tech startup scene (as part of thestartupfactory.tech).

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Aparito selected as a winner of EIT Health’s Bridgehead Global 2020

We were delighted that Aparito was selected as a winner of EIT Health’s Bridgehead Global 2020 programme. The press release is shown below:
Winners of the pilot of EIT Health’s Bridgehead Global 2020 announced

In the pilot year of the Bridgehead Global programme, 21 awarded start-ups and scale-ups seek to expand their businesses beyond Europe. Their products that are ready for sale cover a wide range of technologies from prosthetics or drug testing to medical imaging. These solutions show an enormous impact on citizens that will make their lives easier and improve their overall health. In total 76 applications with a focus on biotech, medtech or digital health were evaluated by three independent jury members. The selected start-ups and scale-ups convinced the jury members in different categories like the innovativeness of their solution, their business model and traction in their home market, and their readiness to expand to beyond-European markets.

Entering a new market presents various challenges ranging from complex regulatory processes to identifying the right collaborators. To overcome these barriers, the EIT Health’s Bridgehead Global programme will support the participating companies by providing them access to experts and enablers. The highly individualised programme will help them reach new markets globally.

The Bridgehead Global programme will connect the chosen start-ups with its established network of so-called Global CATalysers. Supported by these best-in-class accelerators, incubators and clusters, the selected companies are taking their businesses outside of Europe. Contacts to potential customers, distributors as well as guidance on the regulatory and reimbursement systems will facilitate their market penetration in countries like the US, Canada, Brazil, Japan and many more.

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Aparito selected as a winner of the ‘Techforce19’ challenge for COVID-19

In March, DHSC and MHCLG announced that funding would be available to innovators who can find digital ways to support people who need help during the coronavirus (COVID-19) outbreak. This ‘Techforce19’ challenge aims to support those who are particularly vulnerable or isolated as a result of the outbreak and need help, including people who need mental health support or who have social care needs.

NHSX has been working with partners PUBLIC and the AHSN Network to run the fast-track competition for innovators, creating a pool of ready to go technologies that have the potential to be rapidly scaled regionally and nationally.

Following an assessment process, led by Public and working with the AHSN Network, NHSX and representatives from local authority, care and mental health sectors, 18 companies, including Aparito, have been selected and awarded up to £25,000 to take part in a rapid testing phase.
Avoiding contact with others and staying at home is incredibly important, but it’s also incredibly tough. Our technology sector has the talent and ability to help us meet the new challenges this has created, and support unmet need. 

For more information about the Techforce19 challenge click here 

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Aparito joins the fight against COVID-19

Aparito is delighted to announce that we are joining the fight against COVID-19. Using our flagship platform Atom5TM, Aparito will support remote patient monitoring of up to 1,200 cancer patients under the care of Betsi Cadwaladr University Health Board, helping vulnerable patients to stay at home and still receive care and support from their clinicians. #mHealth #wearables #cancer #oncology #Garmin

Aparito is a digital health company with a technology platform (Atom5TM) that supports remote patient monitoring globally. Using patient-generated data in the form of videos, data from Garmin wearable devices, photos and text, data is captured and transferred via the patient’s own smartphone/tablet and made available to clinicians and researchers in real-time. The system is deployed under a Quality Management System for ISO 13485, has Cyber Essential Plus (CE+) in place and operates within the GDPR regulations.

The COVID-19 crisis means that bed capacity in all hospitals is vastly overstretched. All efforts are now focused on both increasing this capacity and reducing the need for patients to be admitted (e.g. through cancelling clinical appointments and day care admissions).

For the oncology population, this is a particularly worrying time. During this COVID pandemic, febrile (feverish) patients have to be assumed as potential virus-positive individuals and kept in isolation. This is because of the risk that if the patient does have COVID-19 they risk contaminating all immunosuppressed patients they may come into contact with during hospital visits.

Thus, such patients should be monitored at home where possible so that low-risk neutropenic patients can more easily and safely be sent home via their Doctor on oral antibiotics with the Aparito digital surveillance solution in place.

At the same time, keeping all patients in hospital which can result in an escalation of care to critical and/or intensive care is not always the right care option for the patient. This means that they may be better supported in their own home with the help of their own family member and the community team. Based on the clinical assessment, physicians may discharge patients with Oxygen therapy and Aparito’s digitally enabled remote patient monitoring solution via our Atom5TM platform and a Garmin wearable device. Garmin wearable devices are not designed or intended to be used as medical devices but can provide health data, including resting heart rate, activity levels and blood oxygen saturation that can be useful in tracking a patient’s health.

We are proud to collaborate with the experienced team at Aparito to help them provide insights for patients facing a higher risk of COVID-19.

Jörn Watzke, Senior Director, Garmin Health BD

Cancer patients – especially those who are immunosuppressed, are feeling particularly vulnerable to serious complications of infection and of side effects of chemotherapy. Our efforts to supply appropriate surveillance at home with minimal impact of patient’s routine will mean that our triage team, led by myself and Dr Nicholas Wreglesworth will be able to provide the best advice and minimise unplanned clinical visits to those really required. This digital solution could moreover pave the path to a more proactive, precise and home-based approach to management of patients receiving anticancer treatment.

Dr Pasquale Innominato, Consultant Oncologist at Ysbyty Gwynedd:

The recent spread of COVID-19 has caused unprecedented demand to our National Health Service (NHS). Providing acute care to COVID-19 without negatively impacting on the care of other conditions will be a major challenge. Over the last five years at Aparito we have deployed our Atom5TMplatform for supporting patient enrolment in clinical trials and research. The solution naturally lends itself to supporting routine clinical care and we are pleased that we can offer this to support Dr Innominato and his colleagues in north Wales.

Dr Elin Haf Davies, Aparito CEO and Founder

Collaboration is key in the fight against COVID-19 which is why Aparito welcomes the opportunity to partner in this endeavour to support remote patient monitoring.

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Aparito and Pulse Infoframe Announce Strategic Partnership

Aparito, a company transforming healthcare by unlocking real-world patient data through mobile apps and wearable devices, and Pulse Infoframe, a leading cloud-based healthcare data insights company delivering a holistic solution to foster collaboration and answer complex research, statistical and economical questions have announced a strategic partnership in rare disease.

The integrated approach will provide an extended offering to users and clients and enable greater insight into the disease burden of patients living with rare diseases. With end-to-end capabilities that link patient-generated data captured remotely via mobile apps and wearables with clinical data, the insight-rich analytics that result will be an invaluable new resource for the life science ecosystem and medicinal products life cycle. 

Our partnership with Pulse Infoframe is an exciting development in our steadfast commitment to deliver unparalleled value to patients and partners. This partnership extends our combined global footprint, which is especially relevant when developing new paths forward for those living with rare diseases. We look forward to growing from strength to strength.

Dr. Elin Haf Davies, Aparito Founder and CEO.

The partnership is addressing high unmet needs in rare disease where significant work remains to be done to increase knowledge of this community. By combining Aparito’s remote data collection approach with Pulse Infoframe’s rich data ecosystem, the solution will generate high quality real‐world evidence while maximizing the efficiency of development programmes. 

This is about excellence and service delivery to the rare disease community. The Aparito/Pulse partnership creates a new, convenient, and seamless pathway to capturing valuable data, from multiple data sources to create a comprehensive view of the impact of medical conditions, in the real-world. The combination of both companies’ expertise can transform the way clinical trials are conducted, leverage the true value of real-world data to accelerate research and collectively can propel science forward and improve the lives of patients and their families. Together, with Aparito, we are eager to serve this important community and address these unmet needs.

Dr. Femida Gwadry-Sridhar, Pulse Infoframe Founder and CEO

About Aparito

Aparito is a global health tech company that brings clinical trials to patients and unlocks real-world data through mobile apps, video assessments & wearable devices. They provide innovative patient-centric clinical trials solutions that integrate specialist medical & regulatory expertise to capture patient data and develop digital endpoints for hybrid and decentralised clinical trials. 

About Pulse Infoframe

Deployed globally and focused on cancer, rare disease, and chronic conditions,  Pulse’s transformative solution healthie™ is a technology platform designed to extract, curate, analyze and disseminate Real-World Data for the purpose of

  • Accelerating scientific research and advancing the delivery of new products to market
  • Optimizing the quality of patient care, while increasing operational efficiencies through workflow management
  • Establishing reimbursement, cost of therapy, and pricing strategies

healthie™ is a platform that encompasses a patient registry and health visualization solution. This GDPR and HIPAA-compliant platform can be described as a collaborative ecosystem that enables the gathering and sharing of information with many partners. Our healthie ecosystem enables patient advocacy, clinicians, patients and researcher partners to increase operational efficiencies by providing insights into complex data that are analyzed and rendered into an easy-to-understand visual display. This intuitive display, provided through a web interface, supports real-time data capture and thus simplifies workflow for building Patient, Natural History and Clinical registries. Pulse Infoframe’s mission is to deliver a holistic solution that answers complex research, statistical and economic questions.

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Developing digital endpoints for the Gaucher community

Aparito Limited, a med-tech company developing innovative digital endpoints to support remote patient data capture has worked extensively with the wider Gaucher community to develop disease specific digital endpoints to support patients. 

Gaucher disease is a lysosomal storage disorder caused by mutations in both copies of the GBA1 gene, which can have a wide range of effects on organs throughout the body. Gaucher disease has three subtypes, which vary by the presence or absence of neurological symptoms, severity of symptoms, age of onset and age at death.

Conveying disease impact and disease-specific hallmarks in young children with devastating rare diseases, such as neuronopathic Gaucher disease, can be challenging. Innovative technology now allows patients and parents to capture outcomes that are important to them with minimal burden and effort. This is an important move towards designing clinical trials that are patient-centric and reducing time spent on hospital visits. 

This innovative technology approach was utilized in one of Aparito’s first studies to evaluate the disease activity of Type 1 and Type 3 Gaucher patients using wearable technology and a disease-specific mobile phone app. The study, which was supported by Sanofi Genzyme and Shire Pharmaceuticals, illustrated that a rich dataset is obtainable and useful for proactive clinical care and for clinical trial outcomes.

Aparito also coordinated the phase one development of a global patient registry for neuronopathic Gaucher disease on behalf of the International Gaucher Alliance in order to increase disease knowledge and understanding, as well as develop new outcome measures and support emerging drugs/ treatments in the pipeline. 

As part of this work, Aparito’s CEO Dr Elin Haf Davies was instrumental in the development of a disease-specific Patient-Reported Outcomes and Caregiver Reported Outcome, which will be undergoing construct validity assessments in the coming months as part of the registry development, presented at the 2020 European Working Group on Gaucher Disease. 

More recently, Aparito, working with Prevail Therapeutics Inc(Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, created a mobile app solution to be used in Prevail’s PROVIDE trial. PROVIDE is an open-label, Phase 1/2, multicenter study to evaluate the safety and efficacy of single-dose PR001 in infants diagnosed with Type 2 Gaucher disease. PR001 is being developed as a potential disease-modifying, single-dose gene therapy for Type 2 Gaucher disease and Parkinson’s disease with GBA1 mutations.

Type 2 Gaucher disease, the most severe form of Gaucher disease, affects infants and toddlers. It causes rapid and irreversible brain damage beginning in the first six months of life; patients typically die by age two. 

The mobile app developed for PROVIDE will capture both clinician and parent-reported assessments of Type 2 Gaucher disease, including feeding and the attainment of developmental milestones. Parents will also use the app to report any seizures that the child may experience. 

We are excited to partner with Aparito and use their technology to broaden how we evaluate the effects of PR001. Type 2 Gaucher disease is the severe, progressive neuronopathic form of Gaucher disease for which there are no approved treatments, and the use of a digital biomarker endpoint in our study will provide a supportive measure of disease burden and severity.

Dr. Eriene Wasef, Medical Director

It’s been a real honour to collaborate with the team at Prevail on this innovative initiative. I have worked with the Gaucher community for over 18 years, and this study brings much needed hope. Knowing the disease so well has helped us to co-develop a digital endpoint that can convey a meaningful and objective measurement without being a burden to patients and families. 

Dr Elin Haf Davies, CEO at Aparito

About Aparito 

Aparito is a global health tech company that brings clinical trials to patients and unlocks real-world data through mobile apps, video assessments & wearable devices.

We provide innovative patient-centric clinical trials solutions that integrate specialist medical & regulatory expertise to capture patient data and develop digital endpoints for hybrid and decentralised clinical trials. 

We do all this through Atom5™, our iOS & Android-compatible web and mobile app platform. Atom5™ captures multiple, high-frequency data points from video, voice, wearables, and electronic Patient Reported Outcomes, then analyses the results in conjunction with our team of data scientists to provide rich, real-time insights to clinical teams. 

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