“Never give up, for that is just the place and time that the tide will turn.”–Harriet Beecher Stowe
As we enter a new decade – one that we believe will see effective therapies become available – the families that form the FSHD Society face new challenges.
This year, we have an unprecedented opportunity to participate in targeted programs designed to accelerate therapies and overcome the obstacles to conducting clinical trials. Additionally, our families around the world will be able to go on record with the FDA through a Voice of the Patient Forum designed to inform and influence the regulatory pathway for promising therapies. This issue of FSHD Advocate lays out The Road to Clinical Trials and many of the activities focused on achieving our collective goal.
Issue Highlights
The Road to Clinical Trials. You might think it would be easy to prove that a treatment for FSHD works. Can’t you just show that a person gets stronger or grows back lost muscles after taking a pink pill? In a word, no. Here’s why.
Member of the team. “My advice for anyone thinking about getting involved in a study is to think of it as an opportunity to be part of something that is so much bigger than oneself.”
The bedrock of clinical trials. This is the very first question a drug company asks when exploring the possibility of developing a drug: Is there a natural history study? That’s how important it is. Many more patients need to enroll in one.
FSHD affects the whole family, but never let it define you. “In many ways, I think our partnership is a way of saying that while this disease can unfairly beat up one of us, together it will not win.”
Read the full issue here.
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