Rare Disease Report
Physicians
Physicians
Patients & Caregivers

Amicus Therapeutics' CabaRare

MARCH 23, 2017



Transcript

Music
Nothing’s holding me back.
Nothing’s holding me back.
 
John Crowley, Chairman and Chief Executive Officer, Amicus Therapeutics
 
Going back to the founding of Amicus and the choosing of the name of the company itself we picked the name amicus — it’s the latin word for friend. And the whole notion is if you had a biotech company that you could start with a blank piece of paper and put people living with rare diseases at the center of that what would it look like? What type of employees would you bring in? Which diseases would you be involved in? What commitments, what promises would you make to the community? And that's what we're trying to do here at Amicus. So just bringing in these people living with rare diseases is just part of the fabric of who we are.
 
Jayne Gershkowitz, Chief Patient Advocate, Amicus Therapeutics
Rare disease day started in Europe with the European Organization for rare disorders and then came to the united states the following year and has just gained momentum ever since.
 
John Crowley
And it really is just that – a celebration of living life despite having challenges with rare diseases no matter what the rare disease may be.
 
Music
Welcome to my world
Won't you come on in
Miracles I guess
Still happen now and then
 
Jayne Gershkowitz
On rare disease day our tendency has always been to bring in people living with diseases that we're not working on sort of broaden our own understanding of rare diseases and that's what we're doing today with CabaRare. We have four individuals coming in each living with the different rare disease and only one of them was with the disease that we currently have a program in.
 
Music
Welcome to my world
 
John Crowley 
Anytime we bring people living with rare diseases into our building, we do it for two reasons. One, to educate us and to make our lives better. And two, for them to see that there is it an organization, a company, like here at Amicus, that truly cares about them and their diseases, even if we're not researching their specific disorder.  We hope to make a dent in the universe in the field of rare diseases.
 
Jayne Gershkowitz 
Something like rare disease day is an opportunity to put all competition aside and say you what are the underlying issues that we need to make people aware of, that we need to fix, that we need to advance in our systems. And of course, that's different in different countries.
 
John Crowley
 So many of these stories are based on how they overcame adversity to live, survive, and thrive. That's true and that'll always be part of it and part of the inspiration. But the other part is actually much more straightforward and that's to show that people living with these severe devastating rare diseases are also just living everyday lives like you and me. They have the same hope, fears, and dreams. And so, in many ways to be able to show that their lives are not only very special, their lives are also very commonplace. I think that's also a very important part of what we've learned and in part, what we want to convey.
 
Music
There’s a reason
There’s a reason
There’s a reason
 



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