August 15, 2019

Yesterday's New York Times featured an editorial about the cost of gene therapy and the recent controversy surrounding Norvatis's gene therapy for SMA.  

Here is co-founder, Maria Kefalas' response to the New York Times.  

Today’s opinion piece on the gene therapy pricin...

October 23, 2016

Not long ago, I found myself speaking to a program administrator at the FDA. It was a classic elevator pitch, I told him all about the breakthroughs in gene therapy for leukodystrophy, specifically, MLD. My voice cracked with emotion recounting how I had recently told...

 Gavin at age 4, before transplant.

Ali and Gavin, post-transplant, Christmas 2014.

 

 

My son Gavin Quimby loves superheroes, particularly Superman, to whom he shares more than a passing resemblance with his dark hair, piercing eyes, and fair complexion. It should not s...

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