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May 11, 2016

TOPIC: Uncategorized

Debra Miller: 2016 Rare Impact Awards Honoree

Posted by Lisa Sencen

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 closest to becoming the first approved treatments for the… Read More

May 11, 2016

TOPIC: Featured News, Patient Stories

Desiree Lyon Howe: 2016 Rare Impact Award Honoree

Posted by Lisa Sencen

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 being saved by this important treatment, she sprang into… Read More

May 8, 2016

TOPIC: Uncategorized, Patients & Members

Happy Mother’s Day from NORD!

Posted by Lisa Sencen
Rare Moms are resilient; fighting the battle against rare diseases day in and day out. Whether they’re a mother dealing with a diagnosis themselves or a mother who is caring for a child with a rare disease, they are strong, passionate, and inspirational women.MothersDayPost_2
Share your mother’s day photos and stories in the… Read More

May 5, 2016

TOPIC: Featured News

Dr. Stephen Cederbaum: 2016 Rare Impact Award Honoree

Posted by Lisa Sencen

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 disorders, and in particular arginase deficiency, in his distinguished… Read More