Global Genes Honors Rare Disease Leaders

'Champions of Hope' announced at 2022 RARE Disease Patient Advocacy Summit

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by Steve Bryson, PhD |

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Global Genes announced the winners of its RARE Champions of Hope awards, recognizing leaders and organizations for their work driving advocacy and innovation in rare diseases, such as cold agglutinin disease (CAD).

As part of the 2022 RARE Disease Patient Advocacy Summit, held Sept. 12–14 in San Diego, California, the rare disease patient advocacy organization acknowledged those who made a significant contribution to patient advocacy, industry, medical care, and research science, as well as recognizing up-and-coming rare disease leaders.

“In a community that is faced with ever-expanding needs and some extraordinary challenges, this year’s recipients were undaunted and forged ahead to accomplish extraordinary things on behalf of people living with rare diseases,” Craig Martin, Global Genes’ CEO, said in a press release.

“We are driven by their examples and inspired to meet the standards they’ve set for us through their determination, ingenuity, resourcefulness and tireless focus on making a meaningful impact,” Martin added.

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Global Genes recognized RARE Champions of Hope in seven areas.

The individual Champion in Advocacy award was given to Adrian Goretzki, founder of the Healthcare Education Institute, a rare disease advocate, for providing treatment to those in need, especially patients crossing the Polish/Ukrainian border in 2022, ensuring access to medical care.

The foundation-based Champion in Advocacy was granted to the Lysosomal Storage Disorders Support Society India (LSDSS India). Lysosomal storage disorders are caused by dysfunction of lysosomes — the cells’ small recycling centers. LSDSS India provided advocacy and awareness at a grassroots level, counseled patients and parents, and delivered therapies to patient homes during the pandemic.

Champion in Industry was awarded to Arthur Levin, PhD, chief scientific officer at Avidity Biosciences, for his decades-long research into the development of RNA-based therapeutics.

Champion in Medical Care and Treatment recognized Yuriy Stepanovskiy, PhD, founder of the Ukrainian Association of Pediatric Immunology, for his help in getting necessary medications to people with rare immunologic and/or autoinflammatory disorders in Ukraine.

Richard Horgan, founder and president of Cure Rare Disease, received the Champion in Science and Technology award for developing therapeutics for ultra-rare neuromuscular diseases.

RARE Rising Star designation was granted to Nell Choi, author, radio DJ, and speaker, for her leadership and advocacy for children in the rare disease community.

The Founder’s Award was given to Joe and Cynthia Lang, founders of Jordan’s Guardian Angels. Their efforts raised millions of dollars for research and treatment of Jordan’s Syndrome, a rare genetic disorder marked by mild-to-severe developmental delay and intellectual disability. Thanks to the Langs’ work, 70 families were flown to Columbia University, in New York, to gather natural history data and collect samples in advance of clinical trials.