Cal's Cupcakes Blog

Inspiration and Hope 

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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