Two Disabled Dudes

Money is a factor in nearly all situations in life - especially when you factor in rare disease or another medical issue.  In this episode the Dudes talk about how money and planning for the future factors into different aspects of their lives - including the age old question "can money buy you love?"

Thanks to the AVROBIO team for a great day of patient advocacy. We feel so honored to be a part of these conferences and always take so much away from them.  And a huge thank you to panelists Jordan, Darren, and Kim.

Gene Therapy treatments have the potential to make a profound impact in rare disease. However, these potentially permanent therapies present unique considerations for any participant in clinical trials.

When we were presented with the opportunity to moderate a panel of people who either participated in gene therapy studies or had extensive knowledge to share on this topic, we jumped on it!

AVROBIO’s vision is to bring personalized gene therapy to the world. They aim to prevent, halt or reverse disease throughout the body with a single dose of gene therapy designed to drive durable expression of therapeutic protein, even in hard-to-reach tissues and organs including brain, muscle, and bone. AVROBIO’s ex vivo lentiviral gene therapy pipeline includes clinical programs in Fabry disease, Gaucher disease type 1 and cystinosis, as well as preclinical programs in Hunter syndrome, Gaucher disease type 3 and Pompe disease. 

For additional information, visit www.avrobio.com 

The Marfan Foundation is currently celebrating 40 years of service to patients and families affected by Marfan Syndrome and other related conditions.

We had the honor of participating in their 36th annual conference with a virtual discussion featuring 4 panelists who have each been significantly affected by rare disease. 

Marfan syndrome is a genetic condition that affects the body’s connective tissue.  Connective tissue helps to hold the body’s cells, organs, and tissues together and also helps to control how the body grows and develops. 

There are several disorders related to Marfan that cause people to struggle with the same or similar physical problems, and anyone affected by these conditions needs an early and accurate diagnosis. Please visit www.marfan.org for more information about Marfan, Loeys Dietz, and Vascular Ehlers Danlos Syndrome.

Listen as Leah, Adrianna, Kristy and Micah share openly about their vastly different experiences but also their shared and wildly optimistic, fulfilling lives, despite the challenges and unpredictability of their conditions.

Old men know a lot. 

Combined, these two Dudes have 37 years of experience living with Friedreich's ataxia/rare disease/disability, and we made some observations along the way.

In this episode, using an idea we found in our friend, Kendall Harvey's column on Friedreich's Ataxia News, we explore what we would say to our newly diagnosed selves.  

It's a very emotional episode and we hope you find value in it.

Anthony DeVergillo describes himself as an optimist.  He sees a problem and inserts himself to find a solution. Specifically in the area of video game accessibility, Anthony is motivated to make an impact with the joystick he invented to allow people with disabilities access to the games they love.  The project is called Overjoyed.

Listen to our interview with Anthony and get motivated to improve the world around you as Sean tries to wrap his head around airplane accessibility.

Links:

Anthony's Interview for The Playability Initiative.

Anthony on social media:

• Twitter
• LinkedIn

Connect with Anthony and others: https://ourodyssey.org/