2018 Top Three Acceptance Speeches.
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Post-show Videos

2018 Top Ten Herbert Pardes Award Winner: Florian Eichler, MD

 

 

Researchers used a lentivirus to infuse a normal copy of the ABCD1 gene into the bone marrow of boys with cerebral adrenoleukodystrophy (ALD), and the corrected protein stopped disease progression. This is the first successful gene therapy treatment to halt a fatal brain disease.

 

2018 Top Ten Distinguished Clinical Research Achievement Award: Timothy McAlindon, DM, MPH

 

 

Painful knee osteoarthritis (OA) affects 10% of the population over 55 years, and is a major cause of work loss, early retirement and joint replacement yet there are few effective treatments and none known to stop it from progressing. The study was done to find out if corticosteroid injections, administered every three months for 2 years into knees with OA and some evidence of inflammation, reduce knee OA cartilage damage, and benefit pain and functional ability in the long-term. However, another important reason to test corticosteroid injections was to see if they damage to joint structures, which has been a longstanding concern.

 

2018 Top Ten Distinguished Clinical Research Achievement Award: Jerry Mendell, MD

 

 

Dr. Mendell’s clinical trial was published in the New England Journal of Medicine (NEJM) in early November 2017 and was the subject of simultaneous commentaries in NEJM and Science. He tested, for the first time in history, intravenous gene replacement therapy for a fatal disease in infants, spinal muscular atrophy. The work was conducted by Dr. Mendell and his multidisciplinary team at Nationwide Children’s Hospital in collaboration with AveXis, Inc. and The Ohio State University College of Medicine.