I favor running analogies even the name of this column pays tribute to the sport. But Im not the first patient advocate to evoke that ancient sport of quicker-than-average terrestrial locomotion. After all, we are racing to treatments because time is critical.

But in the fight against a rare condition like Batten disease, success involves much more than speed. You may be fast, but have you studied the course? Do you know its twists and turns? Do you have a team and support crew, or are you competing alone? Are you ready to take on unexpected obstacles?

Do you have what it takes?

Therapy development isnt for the faint of heart, but patients can and absolutely do make an impact on potentially life-saving treatments and the science and regulatory issues involved. Last month, I joined my mom, Taylors Tale President Sharon King, and Steven Gray, PhD, associate professor at University of Texas Southwestern Medical Center, onstage at the Global Genes RARE Patient Advocacy Summit to discuss that topic.

If you didnt make it to San Diego for the summit this year, I hope you had a chance to tune in to the livestream. If you didnt, or if youd like a recap, following are nine things that we shared about pushing promising work toward the finish line.

Like a marathon, the race to find treatments and cures isnt easy. Few people see the long hours in the lab or the thousands of experiments that dont produce a single breakthrough or correct answer. Miracles happen in science, but results come with a lot of hard work and some luck.

If you want to see a treatment for you or someone you love, you have to accept that it wont be easy. This race is hard. Youll feel winded. Youll get injured. Youll crave water stops. Youll get passed by someone running faster than you. Youll pass someone who doesnt want it as much as you do.

In his new book Chasing My Cure, my friend, David Fajgenbaum, writes, Fear disintegrates. Doubt disorganizes. Hope clears the way, pushes out the horizon, and gives us space to build structures.

Heres what I think David meant by that:

Being invincible in hope means having the courage and the will to take action. You cant trust and wait. You cant assume that someone else will swoop in to save the day. Thats not what hope means.

Instead, you have to look inward. You have to take an active role in transforming hope into reality. Thats hope in action.

Thats how you change the world.

Never doubt that a small group of thoughtful, committed citizens can change the world. Indeed, it is the only thing that ever has. Margaret Mead

***

Note: Batten Disease News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Batten Disease News or its parent company, BioNews Services, and are intended to spark discussion about issues pertaining to Batten disease.

View post:
9 Things We Should Know About the Race to New Treatments - Batten Disease News

Comments are closed.