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Archives for: May 11th, 2016

Debra Miller: 2016 Rare Impact Awards Honoree

Written by Lisa Sencen on May 11, 2016

Debra Miller co-founded CureDuchenne in 2003 with her husband, Paul, after their only son was diagnosed with Duchenne Muscular Dystrophy. With a background in sales and marketing, she leads CureDuchenne and has funded seven research projects that have advanced to human clinical trials, three of which are the… Read More

Desiree Lyon Howe: 2016 Rare Impact Award Honoree

Written by Lisa Sencen on May 11, 2016

Desiree Lyon Howe lived for 10 years in excruciating pain before being diagnosed with Acute Intermittent Porphyria (AIP). She was finally diagnosed at the National Institutes of Health where what would become the first therapy approved as orphan drug, Panhematin®, saved her life.  Upon… Read More

Dr. Stephen Cederbaum: 2016 Rare Impact Award Honoree

Written by Lisa Sencen on May 5, 2016

A medical geneticist and expert on inborn errors of metabolism, Stephen Cederbaum, MD, has been affiliated for many years with the University of California, Los Angeles, as a professor, medical researcher and clinician. He has conducted pioneering research, published widely acclaimed papers and advanced understanding of urea cycle… Read More

Noah Victoria: 2016 Rare Impact Award Honoree

Written by Lisa Sencen on May 5, 2016

Noah Maria Victoria wrote her first poem at the age of 13 or 14 as a homework assignment.  She composed “The Window,” a metaphor for her journey and countless trips to Children’s Hospital and the National Institutes of Health clinical centers.  “My car window has been my companion… Read More