Wall Street Journal Article by Judy Stecker

Pediatric rare disease drug development relies on the commitment, motivation, and tenacity of the parents. BBDF is grateful for our partnership with rare disease families who are advocating for treatments for Batten disease. Congratulations to Batten mom, Judy Stecker, co-founder of Wheeler’s Warriors at BBDF, for articulating some of the challenges faced by rare diseases.


The FDA Could Help Save My Son From a Rare Disease

My eldest son, Wheeler, has a rare genetic disease that will steal his vision, mobility, memory and, ultimately, his life. Every morning when he leaves for preschool, I worry it may be the last time he’ll be able to see my face. 

One in 10 Americans have rare diseases— defined as affecting fewer than 200,000 people in the U.S.—and 95% of these afflictions lack a Food and Drug Administration-approved treatment or therapy. Three in 10 children affected by rare diseases won’t live to see their fifth birthday. Wheeler turns 5 in May. 

When my son was diagnosed with CLN3 juvenile Batten disease at 4 weeks old, research into possible therapies offered hope. That hope is all but gone. The Beyond Batten Disease Foundation’s planned 2023 Phase III clinical trial of a potential treatment is in limbo, seeking funding. (My family supports the foundation.) Earlier this month, Amicus Therapeutics abandoned its pursuit of the only proposed gene therapy. 

These developments are devastating but unsurprising. Given this, the anti-innovation sentiment in Washington and unnecessary bureaucracy at the FDA have made it difficult for companies to justify investments in rare-disease therapies. 


Read the Full Article


My son’s time is running out due to a rare disease. The FDA needs to add more clinical trial flexibility

By Judy Stecker
Feb. 28, 2023

My toddler, Wheeler, will probably not live to adulthood. Juvenile Batten disease — he has the type known as CLN3 — is stealing his childhood. And then this rare disease will steal my child.

Wheeler is missing the DNA needed to recycle a waste product called lipofuscin that his cells naturally produce. As lipofuscin builds up, his condition will get worse, robbing him of his eyesight, his speech, his mobility, and ultimately his life.

Our only hope is finding a treatment to cure, or at least a way to slow down this unrelenting disease. At age 3, Wheeler already has sleep and behavior problems, as well as delays in his speech and fine motor development. He recently began moving closer to the TV and rubbing his eyes, signals that in a matter of months or weeks he may soon live the rest of his life blind.


Read the Full Article

USNA Class of 1986 Fundraiser for Tyler’s Mission

USNA Class of 1986 Fundraiser for Tyler’s Mission

The weekend of 7-10 October marked the 35th Reunion for the Naval Academy Class of 1986. The first event of the weekend was a golf outing at Compass Pointe Golf Course in Pasadena, MD. Seventy-seven classmates gathered to play golf and swap seas stories. But more importantly, the occasion was an opportunity to give back to a classmate in need. Through the selling of various on course items (mulligans, 2′ of string, opportunity to tee off from the fairway on one hole), and a raffle event after the tournament featuring items donated by classmates and local businesses, the Class raised $4862 for Tyler’s Mission in support of the son on classmate Jim Kim. It was a perfect ending to a perfect day. The Class could not be more inspired by the way Tyler lives his life and was honored to make this donation to his fund. Go Tyler! Beat Batten!


Thank You Batten 4 Drew!

Thank You Batten 4 Drew!

A big thank you to Drew, Cara, Mike, and all of the Batten 4 Drew supporters who made a $10,000 donation to Beyond Batten Disease Foundation possible!

Hope on the Green 2021

For the past 12 years we have been blessed with phenomenal weather, enthusiastic golfers, and energetic volunteers. Yesterday was no exception, we gathered once again in celebration of HOPE, with our collective mission in mind… #SaveWill . Thank you to everyone who joined us at our 2021 HOPE on the Green. We look forward to sharing our recap photos online this week!

Thank you to our Photography sponsor:

Hughes & Cozad Orthodontics

BBDF Welcomes Delaney’s Dream

BBDF is proud to partner with Delaney’s Dream from West Virginia and welcomes them to our family of donor advised funds. By connecting affected families and communities, we are able to pool resources and better position ourselves to increase awareness and accelerate research. Delaney lost her battle with juvenile Batten disease on Jan. 21, 2019—less than 4 years after her diagnosis—but her family and friends remain dedicated to raising awareness and finding a cure for juvenile Batten disease. We are grateful for their continued interest and desire to help families and children still living with this disease.

“This quote, ‘It’s the little things that make life BIG,’ describes Delaney perfectly”, Delaney’s mom, Janet, shares. “It didn’t take much to make Delaney happy.”

Read more to learn about Delaney and her family.

BBDF Welcomes Sophia’s Dream

BBDF welcomes Sofia’s Dream to our family of donor advised funds. By connecting affected families and communities, we are able to pool resources and better position ourselves to increase awareness and accelerate research. Sofia, her family and BBDF share the same dream – to find a cure and eradicate juvenile Batten disease – and together we will. We are so grateful to have Sofia’s family as part of our community.

Sofia was diagnosed with juvenile Batten disease (CLN3) in 2015, and despite her vision loss and poor memory she remains eager to explore the world alongside her family, including her twin brother, Max. Click here to learn more about Sofia.

BE Project – Thank You from the Bensons

BE Project – Thank you from the Bensons from Beyond Batten Disease Foundation on Vimeo.

In 2016, after 8 years of funding research for Batten disease, our research team was ready to take the next steps to advance our discovery of a treatment to slow the progression of Batten disease. As you know, The Be Project was launched to raise $6 million in 24 months in order to validate their findings, create patient registries, and advance a host of other necessary steps to prepare for FDA engagement and clinical trials. And, we did just that. WE DID IT!!! We achieved our goal, and we hope you feel so proud and take ownership in this enormous accomplishment!

1 2