The Balancing Act®’s Behind The Mystery on Lifetime Talks Pompe Disease This Tuesday 6/3

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Behind the Mystery of Pompe Disease It’s a devastating medical mystery: random symptoms that present in many common diseases; low awareness and misconceptions among physicians; rare incidence and lack of awareness about the disease’s existence. Chances are you’ve never heard of Pompe disease – a […]

Boston Marathon: Genzyme Runners Supporting ‘Rare’ Cause

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By Anamika Roy Daily News Staff A team of Genzyme employees is running for a “rare” cause on Marathon Monday. In partnership with the National Organization for Rare Disorders, 30 runners make up a team called Running for Rare Diseases (NORD) are raising awareness and […]

“What Are Biomarkers?” a Webinar by the AMDA and Genzyme on April 15th

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Learn about Biomarkers – what they are, why they are important, and what Genzyme is doing to understand Biomarkers in Pompe disease. The event will be hosted by Genzyme and the AMDA, together to support the first Pompe Awareness Day! Date: Tuesday, April 15, 2014 Time: 3 […]

Voices Carry: How History Informs Patient Advocacy in the Biotech Industry

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During this 90-minute discussion presented by the Life Sciences Foundation and MassBio, a panel of biotech leaders will discuss how patient advocacy has played a pivotal role in shaping the biotech industry and how it informs and adds value to new therapies in development today. Moderated by […]

At the Corner of Tragedy and Opportunity

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I met Dr. Steven Gray at a Chapel Hill restaurant in the fall of 2011, three years after he began fighting an ultra-rare disease called giant axonal neuropathy (GAN) in the lab of his mentor and one of the world’s leading gene therapy experts, Dr. […]

Hunting Down My Son’s Killer: One Father’s Journey to His Son’s Ultra-Rare Diagnosis

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I found my son’s killer. It took three years. But we did it. Not quite like this (the picture above). I should clarify one point: my son is very much alive. Yet, my wife Cristina and I have been found responsible for his death. My […]

A Rare Disease Champion For Gaucher Disease

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By Wayne Koberstein, executive editor Rogerio Vivaldi’s experience with rare disease therapies teaches that drug development is never finished until simple and certain access for patients is ensured. Rogerio Vivaldi may be the most unique pharmaceutical executive I have ever met. His background, history in […]