Global Genes, a global advocacy nonprofit working for patients and families of rare and genetic diseases, will help hundreds of patient advocates worldwide, including those for Gaucher disease, with travel stipends up to $100,000 so that they can attend this year’s 5th Annual RARE Patient Advocacy Summit.
The event, hosted by Global Genes, is Sept. 22-23 at the Hyatt Regency Resort & Spa in Huntington Beach, California. Global Genes will also livestream the summit to allow advocates who couldn’t make it to California assist the conference from their homes or workplace. Livestream information will be released later this month.
“The Global Genes scholarship has allowed me to obtain the training, support and inspiration I need to recharge and move forward each year,” Andra Stratton, from Lipodystrophy United, said in a press release. “Because of their support, I have learned my voice matters.”
Right after the RARE Summit, PRA Healthsciences will present the 2016 Tribute to Champions of Hope, an annual event honoring individuals for their outstanding feats of courage in fighting the more than 7,000 rare and genetic diseases estimated to impact about 1 in every 10 people in the United States.
A series of rare and genetic disease organizations will be represented, including cystic fibrosis (CF), Huntington’s disease (HD), Friedreich’s ataxia (FA), spinal muscular atrophy (SMA), juvenile dermatomyositis, atypical HUS, Barth syndrome, Batten’s disease, Cushing’s disease, cystinosis, lipodystrophy, and Gaucher disease (GD), among others.
Gaucher disease is a rare, inherited metabolic disorder in people who don’t produce enough of the enzyme glucocerebrosidase, which results in the accumulation of harmful quantities of lipids (a certain kind of fats), more specifically the glycolipid glucocerebroside, throughout the body.
Some patients develop few or no symptoms, but others may suffer from serious complications. According to the National Organization for Rare Disorders, there are about 6,000 people with Gaucher disease in the United States.
Patients and families can register here for the RARE Patient Advocacy Summit and expect to leave it with actionable next steps to take back to their community, while feeling more empowered to increase individual and collective advocacy efforts. The RARE Summit will feature more than 80 speakers who will present a variety of topics, from funding early research to developing treatments to advocating for specific disease issues.
Speakers attending this year’s summit include:
- Robert Ring, PhD, former chief medical officer of Autism Speaks;
- Anthony Philippakis, MD, PhD, chief data officer at Broad Institute and Venture Partner from Google Ventures;
- Phillip Reilly, MD, JD, author of “Orphan – the Quest to Save Children with Rare Genetic Disorders”;
- Petra Kaufmann, MD, director at the Office of Rare Diseases Research and Division of Clinical Innovation at NCATS, National Institutes of Health;
- Eric Michael David, MD, JD, chief strategy officer EVP at Organovo.
“We have seen incredible growth in requests from patients and their families to participate at our annual summit and we are grateful to be able to offer this support,” said Carrie Ostrea, senior manager of patient advocacy at Global Genes. “This is an important time for Global Genes and our community to unite and provide patients and advocates with the opportunity to learn new skills and connect and share experiences.”
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