Recipients

2017 PAL Awards Grant Recipients

Congratulations to the recipients of the 2017 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.

In the eight years since the program was started, Sanofi Genzyme has awarded close to $810,000.00 to support 52 unique projects proposed by organizations in 28 different countries across the world. As an independent voice in the lysosomal storage disease community, patient organizations play a vital role in providing leadership, resources and access to care and support for patients and their families around the world. Sanofi Genzyme believes supporting their work is critical to optimizing patient care and meeting the ever-growing needs of the LSD community.

In 2017, 36 patient organizations from across the globe submitted proposals for new collaborative and creative activities to support the LSD patient community. Proposals were received from organizations in 24 countries, across a variety of rare disease groups and communities. Programs from six patient organizations were then selected by an Independent External Review Committee. This year’s award recipients come from Ukraine, the United Kingdom, the United States, Romania, France, and New Zealand.

Each proposal represents a unique approach to meeting the needs of the LSD community. Organizations are eager to empower patients, raise the awareness level about LSDs amongst the public, and study trends in rare diseases.

Rare Diseases of Ukraine

Ukraine

“Raising awareness and orphan patients’ advocacy in the regions of Ukraine”

With their grant, Rare Diseases of Ukraine hopes to address unmet needs for adult rare patients. Since many health care providers in the country do not offer care for rare disease patients past the age of 18, their project will focus on advocacy trainings, and creating regional groups of experts - lawyers, doctors, nutritionists - to support these 18+ disease patients. As the project progresses, they plan on publishing information about the rights of adult rare disease patients, and hosting an awareness photo exhibit.

Romanian National Alliance for Rare Diseases

Romania

"Understanding Rare Challenges (URC) in Romania"

The Romanian National Alliance for Rare Diseases project will be an awareness campaign for the public about the specific needs of people living with rare diseases. The group has proposed several activities, including helping Pompe patients form a patient organization, launching a Pompe awareness campaign, organizing a marathon, and sharing patient stories through radio programming and literature. The group plans to share the project outcomes through an existing training course for journalists aimed at improving knowledge of rare disease patients.

Hermansky-Pudlak Syndrome Network

United States

"Individual Research Plan Project"

This organization is proposing an effort to educate patients and families on how to participate in scientific research. Through a steering committee they plan to develop an educational curriculum about informed consent, placebo trials, biospecimen collection, and donation of organs and tissues for research. The project will generate personalized plans for rare patients about how they can participate in scientific research.

Mucopolysaccharide & Related Diseases Society Aust. Ltd.

United Kingdom

"The 'Red Book' research survey for MPS and related disorders"

Using information collected in the Red Book (the UK’s national standard record of a child's health and development) this group will conduct a survey across all patients 25 and under with mucopolysaccharidoses and related disorders, to identify common patterns and trends. The organization hopes that the data might reveal early indicators of the disease or commonalities among patients. Those trends would then be shared with doctors, clinicians, and families, hopefully leading to earlier diagnosis and better outcomes.

Association de Patient de la Maladie de Fabry

France

"FAME : (Fabry: points of view by women)"

The funding from this PAL Award will facilitate a 2-day event for 10 women with Fabry. The event will include a physician-led educational workshop, individual patient interviews with psychologists, and a writing workshop. Post-event, a booklet for patients and HCPs will be published, including information about how women experience Fabry, articles written by the participating psychologists, and patient testimonials. The booklet will be shared in-person and online with psychologists, patients, and physicians.

New Zealand Pompe Network

New Zealand

"New Zealand Pompe Network Inaugural Conference 2018"

The New Zealand Pompe Network will use their PAL Award to fund the first-ever country-wide, multi-organization Pompe conference. Experts from countries with successful care and treatment for Pompe patients will be invited to share information, scientific knowledge, and best practices with New Zealand physicians and patients. Pompe patients will be surveyed in advance of the conference to determine what topics are of the most interest to the community.

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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