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Cheering On Rare Disease Researchers

With pro football fanfare, Uplifting Athletes will celebrate early career scientists later this week by awarding $20,000 grants to support their research into rare conditions.

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Debbie Drane, Senior Vice President for Global Commercial Development and Therapy Area Strategy, celebrates winners of research grants at an Uplifting Athletes Young Investigator Draft.
Debbie Drane, CSL Behring Senior Vice President for Global Commercial Development and Therapy Area Strategy, celebrates the 2023 recipients of the Uplifting Athletes Young Investigator Draft.

Preparations are underway for draft day February 3 at Philadelphia’s Lincoln Financial Field, but these recruits excel in something other than football.

Every year, Uplifting Athletes (UA), a nonprofit organization dedicated to rare disease patients, selects 10 promising scientists in its Young Investigator Draft at the Philadelphia Eagles stadium. Each draftee receives a $20,000 grant to pursue game-changing research into rare conditions.

Leaders with CSL Behring, a longtime supporter of the draft, will attend the high-energy event that showers scientists with fanfare usually reserved for professional athletes. CSL Behring, which makes medicines for rare and serious diseases, is a business unit of CSL, a global biotech company with a campus in nearby King of Prussia, Pennsylvania.

Professional wrestler Joe Spivak, a former defensive lineman with Northwestern University, will introduce this year’s draft class, including Dr. Eleonora D’Ambrosio who is developing a CRISPR-gene editing strategy for valosin-containing protein (VCP) disease. VCP, an adult-onset protein disorder, affects multiple body systems.

Counting the grants awarded to the 2024 draft class, Uplifting Athletes has given more than $1 million in research funds since the Young Investigator Draft launched in 2019. It’s among several programs Uplifting Athletes created with support from college athletes, pro players and patient groups. Other awareness-raising and fundraising programs include Lift for Life and Reps for Rare Diseases.

"Our Young Investigator Draft is a celebration that shines a spotlight on the critical need for rare disease research and honors those who are dedicating their career to maximize that potential," said Rob Long, Executive Director of Uplifting Athletes.

There are thousands of rare diseases and many lack an approved treatment. Rare Disease Day, a day of global awareness raising, is February 29.

Learn more about the Young Investigator Draft at Lincoln Financial

This year’s grant recipients include:

Researcher: Anthony Anzell, Ph.D.
Category of Research: Rare Genetic Disorders
Institution: University of Pittsburgh
Nominated by: 
Cure HHT

Researcher: Eleonora D’Ambrosio, M.D.
Category of Research: Rare Genetic Disorders, Rare Muscular and Neurological Disorders
Institution: Nationwide Children’s Hospital
Nominated by: 
Cure VCP Disease, Inc.

Researcher: Marta Garcia-Forn, Ph.D.
Category of Research: Rare Genetic Disorders
Institution: Icahn School of Medicine at Mount Sinai
Nominated by: 
The DDX3X Foundation

Researcher: Noah Guiberson, Ph.D.
Category of Research: Rare Genetic Disorders, Rare Muscular and Neurological Disorders
Institution: Weill Cornell Medicine
Nominated by: 
STXBP1 Foundation

Researcher: Julian Halmai, Ph.D.
Category of Research: Rare Genetic Disorders, Muscular and Neurological Disorders
Institution: University of California, Davis
Nominated by: 
Foundation for ARID1B Research (FAR)

Researcher: Daniel Kennedy II, Ph.D.
Category of Research: Rare Genetic Disorders, Rare Autoimmune Disorders, Rare Blood Disorders
Institution: Baylor College of Medicine
Nominated by: 
TBRS Community

Researcher: Dorottya Laczko, M.D., Ph.D.
Category of Research: Rare Genetic Disorders, Rare Autoimmune Disorders
Institution: University of Pennsylvania
Nominated by: 
Castleman Disease Collaborative Network

Researcher: Anusha Sivakumar, Ph.D.
Category of Research: Rare Genetic Disorders, Rare Muscular and Neurological Disorders
Institution: University of California, San Diego
Nominated by: 
The Friedreich’s Ataxia Research Alliance

Researcher: Toloo Taghian, Ph.D.
Category of Research: Rare Genetic Disorders
Institution: UMass Chan Medical School
Nominated by: 
National Tay-Sachs & Allied Diseases Association

Researcher: Andrea Wilderman, Ph.D.
Category of Research: Rare Genetic Disorders, Rare Muscular and Neurological Disorders
Institution: Baylor College of Medicine
Nominated by: 
TANGO2 Research Foundation