The Alexion Charitable Foundation Inc

The grant will support the project titled mission/flight coordination: healing journeys for patients battling rare diseases to coordinate free air and ground transportation so patients can access specialized medical care and treatment outside of their geographic area to treat and battle illnesses and other health afflictions including rare and ultra-rare diseases that could be life threatening if the patient does not receive the specialized care that they need. With the consolidation of healthcare entities, we are having more and more patients turn to us for help to access specialized care and treatment that is not available to them in their geographic area. We provide our services for as long and as often as a patient needs our help. Since 1996, we have scheduled over 108,000 patient/passenger missions who have traveled more than 16 million to more than 750 medical facilities. Distance should never be an obstacle for patients to access the medical care they need and deserve.

The grant will support the project titled bridging barriers to care: expanding specialist support for children with rare and complex medical conditions in the uk for the following purpose enhance the diagnostic journey and access to specialised care for children living with rare and complex medical conditions. The support will enable the deployment of a new cohort of roald dahl nurse specialists who focus on children with medical complexities (CMC) over a 12-month period, ensuring that children with CMC and those living with rare diseases receive expert, coordinated care tailored to their unique needs. The program will offer professional development opportunities for existing roald dahl nurses, equipping them with advanced skills to manage the complexities of rare diseases and also support the delivery of educational and skill-building workshops, alongside the distribution of essential resources to families.

The grant will support the project titled improving timely access to care for infants with out-of-range genetic newborn screening (NBS) aims to implement and evaluate a comprehensive educational and social support intervention for families who receive notifications of out-of-range genetic newborn screening (NBS) results. The network's NBS registered nurses (NBS RNS) will educate and support families, conduct an electronic social determinants health (sdoh) assessment, provide families with the option to be connected to a personalized electronic health portal (mychart), and have a telehealth visit with an NBS genetic counselor if indicated. This initiative aims to enhance timely access to care by addressing barriers faced by this vulnerable population.

The grant will support the project titled special day services for young adults living with rare diseases and rare cancers across the uk in 2025 for the following purpose: the project is dedicated to give young adults living with rare diseases and rare cancers, and their caregivers/family much-needed time out from the reality of living with a rare disease. We do this by providing enjoyable and inclusive memory-making experiences which give hope, promote resilience and enhance coping strategies. Our disease agnostic approach means we aim to reach young adults with as wide a range of diseases as possible by promoting our services across medical settings and patient advocacy groups across the uk.

The grant will support the project titled identifying, reaching and supporting caregivers of rare disease patients in diverse and underserved patient populations for the following purpose leading a direct-to-caregiver initiative aimed at expanding access to genetic testing and reducing the time to diagnosis for patients with rare conditions. Can's approach emphasizes the crucial role of caregivers in the diagnostic process and targets zip codes with known healthcare disparities to reach diverse and underserved populations. They partner with 600+ patient advocacy groups (pags) to empower patients and caregivers. By enabling them to easily share their diagnostic journeys through an online chatbot, can aims to simplify the navigation of the testing process.

The grant will support the project titled the first gift of its kind | make-a-wish canadas rare and genetic disease fund for the following purpose: to grant between 250 to 300 wishes to children with rare and genetic diseases this year, a shocking 70% increase from last year.

The grant will support the project titled abc health matters: managing rare diseases and chronic health issues which is an introductory health literacy program that empowers canadians to manage their health more effectively by increasing their confidence when talking about and making decisions regarding health issues with family, friends and health professionals. Renewed support from the alexion charitable foundation would allow us to add additional resources to the new stream of the abc health matters program which was designed to meet the needs of families impacted by rare diseases and chronic health issues. The program will include one new targeted workbook, workshops, outreach to community organizations and health care professionals, a dedicated program landing page, and communications and promotional activities to raise awareness about program offerings, with special attention during october's health literacy month.

The grant will support the project titled mental health & mental wellness programming at seriousfun camps to address and support the mental health needs across our global network of camps, specifically our rare disease camper community. Grant funding will continue to focus on mental health interventions for camper and family programming, mental health professional positions at camp, and mental health trainings and certifications for camp staff.

The grant will support the project titled anticipatory guidance in multiple languages for families of children with severe neurological impairment via CPN'S digital tool. Neurojourney is an educational resource for families and clinicians navigating the ever-evolving needs of a child with severe neurological impairment (sni). It explores the interconnected medical complications common in pediatric neuro-degenerative rare diseases. Designed as an anticipatory guidance tool for parents, the content is organized in phases as they might occur in the illness journey. The resource also includes perspectives on the social and emotional experiences of families as they care for their child, all rooted in the principles of palliative care. Neurojourney is available in both written and audio formats in english, spanish and french. With this funding, neurojourney is also available for affected families speaking portuguese, arabic or chinese soon.

The grant will support the project titled ihope for which aims to revolutionize bridging the genetic medicine gap and provide next-generation care to those who need it most. Using advanced genomic sequencing technology and working with local healthcare providers, genetic alliance can diagnose genetic conditions early and accurately. The primary goal is to provide precision genetic diagnoses to children with previously undiagnosed conditions, enabling targeted treatments that improve quality of life, reduce suffering, and increase survival rates. Establishing a framework for genetic health services in under-resourced areas will ensure long-term sustainability within local healthcare systems.

Support the project titled raregivers global mental health & emotional wellbeing initiative: guiding caregivers and patients through the emotional journeys in rare, chronic & complex disease for the purpose of subsidized fees for raregivers wellness retreats, free support groups and public webinars, ensuring inclusivity and global reach. Raregivers retreats, webinars and support groups are designed to create a supportive community, fostering resilience and empowerment among rare disease caregivers.

The grant will support the project titled a new hope | wellbeing in rare for that aims to create a mental health and education program to support teenagers and young adults who are potential caregivers and future patients in the rare autosomal dominant disease space. Participants will have access to genetic testing and counseling services, monthly check-ins with a social worker, and multiple forms of education and peer engagement, including social media content, webinars, and peer support groups. This will provide essential support and prepare participants to make decisions about their future career, personal health, family planning, and care needs.