The AMDA's Hopes for the Future

Robert H. Goddard said: "It is difficult to say what is impossible, for the dreams of yesterday are the hopes of today and the reality of tomorrow."

Twenty-five years ago, when the AMDA was founded, our hope for the Pompe community was a treatment. Today, that treatment is a reality.

However, just because one hope is realized does not mean that we stop. We continue to hope and dream for a better future for the Pompe Community. Gene therapy, next generation ERTs, muscle regeneration and stem cell therapy are among just a few Hopes for the Future that the AMDA has today.

But as I was writing this I realized that my hopes vary for different segments of the community. For those who have not yet been born, we hope for a day where newborn screening is a reality. Where Pompe disease can be treated or cured before any symptoms occur. A future where parents do not constantly fear for their child's life. So the AMDA's hope for the future of patients who have not yet been born is that newborn screening will be a reality for them. That they will be able to access treatment or a cure before they have to live with the inevitable symptoms of Pompe.

For those who have yet to be diagnosed, we hope for a future where diagnosis is easier. A future where it doesn't take decades of searching for answers to receive a diagnosis. We know that early diagnosis is the key to the best outcomes for patients, and yet the path to diagnosis is still fraught with difficulties. So, the AMDA hopes and strives for a future where that is not the case.

Finally, for those who have already been diagnosed, the AMDA hopes that all patients around the world will have access to current treatments. Even though the current treatment has been commercially available in most countries since 2006, it is not available everywhere. The AMDA hopes that one day treatment across the globe will be a reality. We also hope that future treatments will be better. For some that means a treatment that is more convenient. For others it means a treatment that will repair lost muscle. Regardless of how you or your family define "better," the AMDA hopes that your definition will become a reality in the future.

Ultimately, only time will tell which hopes become tomorrow's realities. But the AMDA believes one thing above all—in order to make any of our Hopes for the Future a reality, we must all work together. The patient community, the medical community and industry all have important roles to play—they are all different, but all equally important. However, in the last twenty-five years one thing has remained very clear—it is by working together that great things have happened for our Community. Together We Are Strong, has never been more true. Here is to all of our Hopes for the Future. Happy International Pompe Day!

Tiffany House
AMDA President