Editor's Note : Brian Wamhoff, PhD, is the Cofounder and Vice President of Research and Development, for HemoShear Therapeutics Charlottesville, Virginia
Marshall L. Summar, MD: Hi. I'm Dr Marshall Summar. I'm here at the NORD Summit in Washington, DC, and I have the great privilege of speaking with my friend, Brian Wamhoff, from HemoShear Therapeutics. It's a biotechnology company in Charlottesville, Virginia. We are going to have a conversation today about some new technology developments in the field of rare diseases.
Brian, thanks for taking the time to be here today.
Brian Wamhoff, PhD: Thanks, Marshall.
Challenges to Understanding Rare Diseases
Dr Summar: We've been working together for about a year on a joint project between Children's National Medical Center and HemoShear on rare diseases. Do you want to describe what we've got going on?
Dr Wamhoff: It's pretty exciting. We're developing therapeutics right now for children with organic acidemias. One of the challenges is how to get cells from kids who are alive, particularly from their liver, so that we can understand the biology. As you know, very rarely do mouse models mimic rare human genetic diseases. The bigger challenge is that once you get the cells out of a patient, they do weird things.
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COMMENTARY
The Future Is Now: Rare Diseases in a Dish
Marshall L. Summar, MD
DisclosuresNovember 17, 2015
Editorial Collaboration
Medscape &
Editor's Note : Brian Wamhoff, PhD, is the Cofounder and Vice President of Research and Development, for HemoShear Therapeutics Charlottesville, Virginia
Marshall L. Summar, MD: Hi. I'm Dr Marshall Summar. I'm here at the NORD Summit in Washington, DC, and I have the great privilege of speaking with my friend, Brian Wamhoff, from HemoShear Therapeutics. It's a biotechnology company in Charlottesville, Virginia. We are going to have a conversation today about some new technology developments in the field of rare diseases.
Brian, thanks for taking the time to be here today.
Brian Wamhoff, PhD: Thanks, Marshall.
Challenges to Understanding Rare Diseases
Dr Summar: We've been working together for about a year on a joint project between Children's National Medical Center and HemoShear on rare diseases. Do you want to describe what we've got going on?
Dr Wamhoff: It's pretty exciting. We're developing therapeutics right now for children with organic acidemias. One of the challenges is how to get cells from kids who are alive, particularly from their liver, so that we can understand the biology. As you know, very rarely do mouse models mimic rare human genetic diseases. The bigger challenge is that once you get the cells out of a patient, they do weird things.
Cite this: The Future Is Now: Rare Diseases in a Dish - Medscape - Nov 17, 2015.
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Authors and Disclosures
Authors and Disclosures
Author
Marshall L. Summar, MD
Chief, Division of Genetics and Metabolism; Margaret O'Malley Chair of Molecular Genetics, Children's National Medical Center, Washington, DC
Disclosure: Marshall L. Summar, MD, has disclosed no relevant financial relationships.