Recipients

2016 PAL Awards Grant Recipients

Patient organizations from around the world responded to the 6th Annual PAL Awards program in 2016, submitting proposals for new collaborative and creative activities to support the LSD patient community. Out of 27 applicants, six patient organizations were selected by an Independent External Review Committee.

Proposals were received from patient organizations in 15 countries, across a variety of rare disease groups and disease communities. The successful applicants came from Vietnam, Poland, United Kingdom, Croatia, Brazil, and the United States/India.

The Independent External Review Committee spent hours carefully reviewing each application and examining the merits of each project. Ideas for new collaborative and innovative projects ranged from developing online platforms to educate medical students on the symptoms of lysosomal storage disorders to developing eLearning modules to train and inform patient advocate affiliates and patients about genetic sequencing. Each proposal was unique and sought to meet the needs of the LSD community.

Vietnam Rare Disease Network (VRDN) and Vietnam National Hospital of Pediatrics (NHP)

Vietnam

“Hear Our Stories”

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. 

Polish Association of Families Affected by Gaucher Disease, Polish Association of Families Affected by Fabry Disease, and The Polish Pompe Association

Poland | www.fabry.org.pl

“Storage”

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.

The Association For Glycogen Storage Disease and The Royal College of General Practitioners (RCGP)

United Kingdom | www.agsd.org.uk

“eLearning Module for General Practitioners”

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.

Croatian Alliance for Rare Diseases and National Organization for Rare Diseases Serbia

Croatia | www.rijetke-bolesti.hr

“Little Big Signs of Diagnosis”

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.

Associação Catarinense de Pacientes e Amigos de Gaucher and Associação Gaúcha de Fabry

Brazil | www.acpaggaucher.org

“Disease Awareness and Education Through Health Centers”

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.

Rare Genomics Institute (RG) and Organization for Rare Disease India (ORDI)

The United States and India | www.raregenomics.org

“Rare Disease Patient Advocate Teaching Modules”

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.

Expression of Hope

expressionofhope

A Global Program of Awareness and Inspiration Featuring Works of Art by the Lysosomal Storage Disorder Community

Learn more

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