Recipients

2013 PAL Awards Grant Recipients

Fundación de Pacientes Lisosomales de Chile (FELCH)

Fundación de Pacientes Lisosomales de Chile (FELCH)

Chile

“Healthcare Student Awareness Program”

Through this project, FELCH seeks to increase awareness of Lysosomal Storage Diseases in Chile through an education and outreach program among university students. This national educational campaign will inform and empower future healthcare professionals through seminars and materials distributed by patients and physicians knowledgeable about LSDs. By empowering the next generation of medical professionals, FELCH hopes to ultimately improve diagnosis and treatment of Pompe, Gaucher, MPS I, and Fabry diseases.

China Dolls Center for Rare Disorders

China | www.chinadolls.org.cn

“Our World: Rare and Real – Traveling Documentary Photography Exhibit”

The Our World Photography Project seeks to raise awareness of rare diseases through a traveling, nationwide photography exhibit featuring individuals living with Lysosomal Storage Disorders. Through this exhibit, China Dolls aims to increase understanding and awareness of the impact LSDs have on individual lives, and to foster policies that protect the basic rights of all individuals living with a rare disease to access medical treatment, education, and employment. The exhibit will tour major city centers including Beijing, Shanghai, Shenzhen, Guangzhou, and Xi’an and will include media outreach throughout the tour.

Gaucher Association Denmark on Behalf of a Collaboration of Six Danish Patient Associations

Denmark

“Together We Can Be Stronger” Family Conference

Six Danish patient organizations will cohost the first-ever family conference for patients and their families living with metabolic disorders in Denmark. Collectively, these associations focus on: Congenital Disorders of Glycosylation Syndrome, Fabry Disease, Gaucher Disease, MCAD Deficiency, Protein Degradation Defect Diseases, and Wilson Disease. In addition, the collaborative expects to involve other families dealing with rare metabolic disorders that don’t have a patient organization to support their needs. Organizers hope to reduce the sense of isolation for families, engender a greater sense of community, and generate momentum for collective action in the future through this innovative conference.

Foundation for Patients with Fabry Disease

Hungary

“Fabry Diagnosis and Monitoring System” Diagnostic Standardization

Through this project, the Foundation for Patients with Fabry Disease seeks to improve access to medical treatment for patients by developing a Fabry Diagnosis and Monitoring System to be utilized throughout the country. Because physical examinations are conducted at many sites nationwide and physicians’ knowledge and experience with Fabry disease can vary widely, the project intends to create a clear, objective examination protocol that will ultimately improve treatment decisions by the government health care agency. After a comprehensive research and development phase, a pilot program will be launched and the project will culminate with a publication, with the ultimate goal of national adoption.

Philippine Society for Orphan Disorders (PSOD)

Philippine Society for Orphan Disorders (PSOD)

Philippines | www.psod.org.ph/

“I Can Be Like You” Animated Video

Through this PAL project, PSOD will expand its national rare disease awareness campaign through the creation of a first-ever animated film to be used in social media format. This film will be treated as a children’s story highlighting a little girl’s journey to obtain the correct diagnosis and the importance of getting the proper care once the diagnosis has been made. Using this animated format, PSOD will simplify complex medical terms to improve understanding and increase social sharing via the Internet. PSOD hopes to use the film to accelerate their broader awareness and advocacy campaigns to improve access to sustainable treatment for rare disease patients throughout the Philippines.

LSD Collaborative

LSD Collaborative

United Kingdom | www.facebook.com/UkLsdPatientOrganisationCollaborative

“If you hear hooves it may be a zebra……” Medical Student Empowerment Project

The UK LSD Collaborative – a collaborative of six rare disease patient organizations -- seeks to improve diagnosis and treatment of rare diseases in the U.K. by educating and empowering the next generation of health care providers. Together, the organizations of the UK LSD Collaborative represent the Batten, Gaucher, Fabry, MPS, Niemann-Pick, Pompe and Krabbe disease patient communities. Through their PAL project, the Collaborative will partner with 5th year medical students from the Barts London School of Medicine and Dentistry, as well as the student-created London Society for Rare Diseases to support the development of rare disease societies across the London Medical Schools. Other activities include the development of a website, toolkit, and speakers bureau.

Batten Disease Support and Research Association (BDSRA)

Batten Disease Support and Research Association (BDSRA)

United States | www.bdsra.org/

“Multicultural Clinical Practice Guidelines for Batten Disease” Guideline Development

The BDSRA seeks to improve the diagnostic efficacy and disease management for Batten disease patients by producing and publishing clinical practice guidelines for medical professionals. These guidelines would utilize existing clinical experience and expertise of the Batten Disease Centers of Excellence in the United States, Serbia, and Argentina. Ultimately, these guidelines will be available in multiple languages to support global care. The project will provide physicians with clinical guidelines to inform medical care decisions, advance linkages between LSD Centers of Excellence and community physicians and pediatricians, and provide a multicultural template for LSD clinical practice guidelines derived from known experts in Batten disease.

National Tay-Sachs and Allied Diseases Association (NTSAD)

National Tay-Sachs and Allied Diseases Association (NTSAD)

United States | www.ntsad.org/

“Rare Siblings Project” Video Series

This project seeks to create a web based video series designed to support siblings of children affected with life limiting illness, as well as those living with complex and chronic conditions. Specifically, the project will look to support children between the ages of 6 and 18 years of age. Intended outcomes of this project include reducing feelings of isolation often experienced by siblings, provide siblings with a forum for self-expression, provide parents with insights into the sibling perspective, and develop a community of sibling support. Videos will ultimately be promoted using social media channels such as You Tube and Facebook and will be accessible by the broader rare disease community.

The Patient Advocacy Leadership Award is a rare opportunity for organizations to access funds that help them think about the message and the content that they want to get out there that is particular to them, that they think will make a difference in the lives of other."
— Blyth Lord from the National Tay-Sachs and Allied Diseases Association

Expression of Hope

expressionofhope

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

Learn more

back to top