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BATTEN DISEASE REPRESENTED AMONG FDA PATIENT REPRESENTATIVES

ucm475291The U.S. Food and Drug Administration’s (FDA) patient representative program has existed since 1999 and is integral to fulfilling FDA’s strong commitment to ensure that the needs and choices of patients – as well as their families, caregivers, and advocates – are incorporated in ever greater ways in the work they do.

Recently, the FDA met with 21 inspirational patients and patient caregivers who have made the extraordinary commitment to become FDA patient representatives. These volunteers were in Washington to participate in a two-day Patient Representative Workshop so they can receive training that will allow them to help the FDA meet its critical responsibility of guiding the development and evaluation of safe and effective medical products.

Among these representatives were caregivers who have a personal experience with Batten disease. Sadly, each lost a young son to the disease. But in the face of this tragedy, these two mothers have advocated tirelessly to find a cure for this disease and worked to educate other parents, much like the way that our foundation was started. Here at BBDF we believe in finding hope in the face of this disease. We believe in working tirelessly to find a treatment for Batten disease and other rare diseases in the process. To read the full article on the FDA’s blog click HERE!

RELATED LINKS

http://blogs.fda.gov/fdavoice/index.php/2016/07/the-unique-voices-of-our-patient-representatives/

http://beyondbatten.org/understanding-batten/what-is-batten/

http://beyondbatten.org/events/family-night/

Batten disease on Good Morning America

 

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Taylor’s Law: North Carolina’s rare disease advisory council

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Taylor King, 17, and her mother Sharon.

On July 29, 2015, house bill 823, An Act Establishing the Advisory Council on Rare Diseases within the School of Medicine at UNC Chapel Hill, was ratified by the North Carolina General Assembly. The Governor signed this legislation on August 5, 2015.  The legislation received bi-partisan support and passed unanimously through the NC House and Senate. Now, a year later,Gov. Pat McCrory following a unanimous vote by the N.C. legislature has renamed the legislation Taylor’s Law in honor of Taylor King, 17, who suffers from infantile Batten disease. To read the article click HERE.

Taylor’s Tale, a 501c3 nonprofit organization, was founded in 2007 by family and friends of Taylor King of Charlotte, North Carolina, who was diagnosed with infantile Batten disease in 2006 at age 7. Since then the organization has been a leader in rare disease advocacy efforts.

Taylor’s Tale President Sharon King was the catalyst for this bill, which was championed by N C. Representative Becky Carney (D-Mecklenburg). Sharon first presented the idea to state legislators at a breakfast sponsored by Taylor’s Tale in late March 2015. After the bill was filed on April 14, it sailed through the House and Senate in just over three months, passing unanimously at each stage. House Bill 823 established an advisory council on rare diseases within the School of Medicine of the University of North Carolina at Chapel Hill.

This council will act as advisors on research, diagnosis, treatment and education relating to rare diseases. Appointed members will include:

  • A physician with experience researching, diagnosing or treating rare diseases
  • A medical researcher with rare disease research experience
  • A registered nurse or advanced practice registered nurse with experience treating rare diseases
  • One rare disease survivor
  • One rare disease foundation representative
  • One representative from each academic research institution in North Carolina that receives any grant funding for any rare disease research

RELATED LINKS

http://www.charlotteobserver.com/living/health-family/article87232977.html

http://taylorstale.org/advocacy-efforts/

http://www.med.unc.edu/ncrarediseaseinstitute/about

https://battenblog.com/2015/10/01/my-perspective-laura-king-edwards-8/

How to Apply for Social Security Benefits for Your Child With Batten Disease

by Bryan Mac Murray, Outreach Specialist, Social Security Disability Help

Batten disease is a genetic disease which can devastate a family. After receiving a Batten diagnosis, parents and families are affected in all areas of life. The care, medication, and equipment required for keeping your child healthy is incredibly expensive.

This can bring a massive financial burden upon a family. A child with Batten disease may need round-the-clock care, and one parent may need to leave work to provide that care. Medicine is expensive, and necessary supplemental care has a high cost. Social Security disability benefits are available to help families manage the costs caused by their child’s disease.

Read the full article

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Batten disease on Good Morning America

BBDF is excited to have the international spotlight on Batten disease last week. We have worked with Dr. Pearce in the past and agree that emerging technologies like stem cell therapy may be important in the fight against Batten disease. For this reason we’ve been funding the development of iPS stem cells with the New York Stem Cell Foundation over the past few years, as well as many other tools like it. As Dr. Pearce mentioned, stem cell therapy is probably still several years away. We are also closely following and in some cases supporting other new technologies/research initiatives like gene therapy and gene skipping, which are currently being applied to Batten Disease. Some are showing promising results. That is why the medical breakthrough discovered by our research funded at Texas Children’s Hospital is so important. The treatment slows the disease progression, buying precious time for more research. We will continue executing our 24 month, $6 million plan to validate the treatment option and to enlist the help of regulatory experts to prepare a package to present to the FDA for clinical trial approval. Your help comes at a crucial time and with your support we will continue advancing all treatment options to get it to the kids who need it most.

Read more about it here.

Beyond Batten Disease Foundation grantee, Andrea Ballabio, MD, is awarded the 2016 Louis-Jeantet Prize for Medicine

Dr Andrea Ballabio, MD, Beyond Batten Disease Foundation grantee at the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s Hospital in Houston Texas and founder and director of the Telethon Institute of Genetics and Medicine (TIGEM) in Naples Italy, is awarded the 2016 Louis-Jeantet Prize for Medicine. Established in Geneva, Switzerland. The Louis-Jeantet Foundation furthers the cause of medicine by awarding $723,000 to scientists who make discoveries considered to be pivotal to the treatment of disease.Drs Ballabio and Sardiello’s identification of a master gene that controls lysosomal function and promotes intracellular clearance of accumulating pathological materials revealed a previously unknown biological pathway and provided a powerful tool to modulate lysosomal function to treat juvenile Batten and other diseases. Based on this discovery, BBDF, provided Drs Ballabio and Sardiello over $4 million, enabling them to move to the largest medical center in the world, and more than double our investment by attracting government, pharmaceutical and nonprofit funding, and focus on developing a treatment for juvenile Batten disease. View the press release here.

Mishawaka Family Honors National Rare Disease Day

By Kofo Lasaki | Feb 28, 2016 | NewsCenter 16

Mishawaka- Over the years NewsCenter 16 has shared with you the story of Tyler Allman and how the Mishawaka community has come together to support him and his battle with Batten Disease.

Monday is National Rare Disease Day and the Allman family wants to bring awareness to the thousands of uncommon disease that will affect 1 out of every 10 Americans.

The Allman family says you’re given what you’re given for a reason.

And for whatever reason they were given Tyler, their 14-year-old son who loves life and brings a smile to everybody’s face.

“We have hard days where we look at each other and think why is this happening, not just to us, but even Tyler,” said,Tyler’s Mom, Heather Allman.

Tyler was diagnosed with a rare genetic nervous systemdisorder called Batten Disease.

He’s lost his ability to see and he’s been bound to a wheelchair since he was 11.

“He’ll never have kids; I’ll never have grandchildren from him. None of those things he’ll ever have because today will be better than tomorrow,” said Tyler’s Mom.

Tyler’s brain is slowly shutting down and each day is a little worse than day before.

The Allman’s know time isn’t on their side, so they enjoy each and every moment, but it’s hard not to wonder what could’ve been.

“From one year to the next to see him kind of regress, and not be the natural athlete I know he could be without the disease, yeah it’s tough,” said Tyler’s Dad, Royer Allman.

But tough times are made a little easier knowing they have the support of the community.

That said, for Rare Disease Day you could wear your team Tyler shirts or the colors if you don’t have one.

A small gesture by Tyler’s school makes all the difference in the world.

“It’s those moment where you go in the corner and cry because you’re just so blessed that people do this for you without asking you or making you have to do something for it,” said, Heather Allman.

Which is why they hope to pay it all forward one day.

“We will always try and do him good and always fight for a cure and always fight for other kids and adults that suffer from rare diseases. That will be our attention some day and it will all be because of this child who has no clue what his future holds, but has touched us so deeply,” said Tyler’s Mom, Heather Allman.

On Monday, Tyler’s classmates, along with the entire school at Hums Elementary will be wearing their team Tyler shirts.

Dr. Tammy Kielian: Finding a Cure for Her Niece

Please view this video describing Dr. Tammy Kielian’s work at the University of Nebraska. Dr. Kielian is spearheading a program to treat children with juvenile Batten disease, like her niece Olivia, with two different and potentially beneficial medications. Just like BBDF-funded investigators at Texas Children’s Hospital, Dr. Kielian is working as quickly as possible to bring her ideas to the Federal Drug Administration (FDA) for approval to conduct a clinical trial.

View video here.