In December 2016, we announced the recipients of our 6th annual Patient Advocacy Leadership (PAL) Awards. The global PAL Awards program supports projects by non-profit organizations that serve patients living with lysosomal storage disorders (LSDs), a group of rare, inherited disorders that can cause progressive and debilitating health problems.
Since 2011, through the PAL Awards program, we have recognized 46 organizations around the world supporting disease awareness, community mobilization, non-profit development and good governance activities.
Once again, in 2016, patient organizations from across the globe responded to the PAL Awards program, submitting proposals for new collaborative and creative activities to support the LSD patient community. Proposals were received from patient organizations in fifteen countries, across a variety of rare disease groups and disease communities. Out of 27 applicants, six patient organizations were selected by an Independent External Review Committee. The 2016 award recipients came from Vietnam, Poland, United Kingdom, Croatia, Brazil, and the United States/India collaboration.
Ideas for new collaborative and innovative projects ranged from developing online platforms to educate medical students on the symptoms of LSDs to developing eLearning modules to train and inform patient advocate affiliates and patients about genetic sequencing. Each proposal represented a unique approach to meeting the needs of the LSD community.
We are pleased to announce the following 2016 PAL Award recipients:
Polish Association of Families Affected by Gaucher Disease, Polish Association of Families Affected by Fabry Disease, and Polish Pompe Association (Poland)
The Polish Association of Families Affected by Gaucher Disease, the Polish Association of Families Affected by Fabry Disease and the Polish Pompe Association are joining forces to create an online platform called “Storage” that provides comprehensive education about lysosomal storage disorders and an interactive forum for patients to connect with one another. As the project progresses, the platform will be used to gather and assess the needs of the LSD community to further inform Poland’s patient organizations’ programs and initiatives.
Croatian Alliance for Rare Diseases and National Organization for Rare Disease of Serbia (Croatia)
A rare disease patient’s diagnostic journey is often long, complex and filled with misdiagnoses. Aimed at reducing the length of time to diagnosis, the Croatian Alliance for Rare Diseases and the National Organization for Rare Disease of Serbia are collaborating on a project called “Little Big Signs of Diagnosis,” an educational online platform for medical students. The platform will include videos of patients and their caregivers describing the first and most important symptoms of their disease, in an effort to raise awareness among medical students and better prepare them to correctly diagnosis LSDs in the future.
Rare Genomics Institute and Organization for Rare Disease India (United States and India)
The genes that cause many LSDs have been identified, often making genetic sequencing a useful diagnostic tool. However, in order for genetic sequencing to be performed, patient advocate associates, who help patients navigate the process, must be properly trained, and patients and their caregivers must be properly informed. In an effort to strengthen and optimize genetic sequencing support functions, the Rare Genomics Institute in the United States and the Organization for Rare Disease India are working together to create training modules and educational resources for patient advocate associates, patients, caregivers and physicians.
Vietnam Rare Disease Network and the Vietnam National Hospital of Pediatrics (Vietnam)
Increasing knowledge and reducing stigma around rare diseases are two big challenges for advocacy organizations. To address both issues, the Vietnam Rare Disease Network, in collaboration with the Vietnam National Hospital of Pediatrics, is publishing a book to provide disease information about specific LSDs, as well as share the stories of 22 rare disease patients and their families. It will be the first rare disease awareness book in Vietnam and can also serve as a reference manual for healthcare students.
The Association for Glycogen Storage Disease and Royal College of General Practitioners (UK)
For many rare disease patients, their diagnostic odysseys often begin with a general practitioner who does not recognize diagnostic symptoms or refer them to the appropriate specialist. The Association for Glycogen Storage Disease is working with the Royal College of General Practitioners to educate general practitioners in the UK on the early signs and symptoms of Pompe disease and other glycogen storage diseases. They are developing concise visual e-learning modules to be featured on the website of the Royal College of General Practitioners, an organization with more than 52,000 general practitioner members.
Associação Catarinense de Pacientes e Amigos de Gaucher and Associação Gaúcha de Fabry (Brazil)
Many health professionals are not specifically trained on how to care for patients living with rare and chronic health issues. To address this knowledge gap in Brazil, the Associação Catarinense de Pacientes e Amigos de Gaucher and the Associação Gaúcha de Fabry are collaborating to provide informational materials and trainings at community health centers, targeting doctors, nurses, receptionists, psychologists, and social workers. The trainings aim to help healthcare professionals identify rare disease patients early in their visit to a health center, so they can offer personalized care that addresses the particular needs of their disease and reduces the risk of inappropriate procedures.
For more information about the PAL Awards program, visit www.PALAwards.com.