August 2020                                                                                        Volume 31, Issue 8
BDSRA Advocacy Training
Upcoming Webinars 

The Patient Experience: Introduction to Drug Development and Clinical Trials
Tuesday, Sep 15, 10:00 AM EDT
Cost: Free

"Of all the rare diseases in the world only 5% have a treatment - meaning 95% do not!
According to the U.S. National Institutes of Health (NIH), 85% of patients are unaware of clinical trials as an option at the time of their diagnosis. This is particularly relevant when it comes to rare diseases.

Patients and families dealing with rare diseases will often hear about clinical trials, without understanding what the process entails. A lack of awareness and understanding is recognized as being one of the greatest barriers to clinical trial participation by patients.

The purpose of this series of two webinars is to explain the development process of new drugs and what it means to participate in a clinical trial. Our goal is to help educate patients and their families who are dealing with rare disease, providing useful information on what is involved in clinical research."

The Patient Experience: What is it Like Participating in a Clinical Trial?
Tuesday, Oct 20, 10:00 AM EDT
Cost: Free

"After exploring the landscape of drug development in our first webinar, our second webinar will focus on what it means to participate in a clinical trial, with a focus on the patient's perspective.

It is crucial for patients and their families to understand what is involved, in addition to being aware of their rights and obligations, if they so choose to participate in a clinical trial."
ForeBatten Butterfly Art Contest


The contest celebrates ForeBatten Foundation's butterfly logo which represents endurance, change, hope, and life. 

Artists of all ages are encouraged to participate and any type of artistic creation may be submitted.

To see all of the submissions or to enter yourself, clink on the link in the comments.
Winners will be announced November 15th and will receive their art displayed as a one-of-a-kind quilted wall hanging. Invite your family, friends, co-workers, and classmates to participate!

Learn more, see submitted art, and submit your own here!

A Toolkit: Helping Your Child Wear a Mask During COVID-19

As schools and businesses start to open up we wanted to share this wonderful resource from the University of Rochester on Helping Your Child Wear a Mask During COVID-19. The toolkit includes tips for helping your child understand why masks are important, places you can buy masks, and other great resources for the children in your life.

You can download the toolkit here.
Research News 

In the last month we have had a number of exciting updates in the clinical research space. 

Abeona Therapeutics:
"Abeona Therapeutics and Taysha Gene Therapies Enter into Licensing and Inventory Purchase Agreements for ABO-202, a Clinical Stage, Novel, One-time Gene Therapy for CLN1 Disease"
Learn More Here: https://investors.abeonatherapeutics.com/press-releases/detail/189/abeona-therapeutics-and-taysha-gene-therapies-enter-into

University of Rochester Batten Center:
"The URBC is recruiting individuals with CLN3 disease to participate remotely in a new study! Visits will occur once every six months for three years."
Learn More Here: https://www.facebook.com/URMCBattenCenter/photos/a.113796095494138/1438105173063217/

Neurogene
"Neurogene's latest effort to help patients and families affected by rare neurological diseases, including Batten disease, subtype CLN7. Neurogene has received Orphan Drug Designation (ODD) for their CLN7 gene therapy program.
Receiving Orphan Drug Designation enables Neurogene to continue to work closely with the FDA moving through the approval process in an accelerated manner. This opportunity provides Neurogene with a potential decreased wait-time for drug approval, while getting a quality and safe treatment to patients faster. "

RegenxBio
"RegenxBio has recently announced a new program, RGX-381, for the treatment of vision loss in patients with CLN2 disease. We are committed to continuously evaluating our portfolio of gene therapy candidates, with innovation and patient needs in mind. Today, we have announced the expansion of our pipeline to include RGX-381 for the treatment of ocular manifestations of CLN2 disease, also known as Batten disease. This will complement our existing RGX-181 program for the treatment of CLN2 disease in the central nervous system. I look forward to providing additional details and updates for RGX-381 and RGX-181 in the coming months."

If you have questions please reach out to [email protected]



This month we have new Facebook fundraisers organized by:

We are so excited each day to log in and see the list of your names growing. Thank you for sharing and supporting our mission on social media! You can visit each fundraiser by clicking on the names above. 
In Loving Memory 

Remember with us those we have lost to Batten disease.  It's in their honor and memory that we work every day to build a brighter future for families.

Severide Welch
8/26/2014 - 2/24/2020

Luka Mamula 
6/3/2020 - 8/19/2020

Emeli Lozano
8/9/2020
Your gift makes all the difference in the lives of families. 

You can view recent donations to BDSRA here.
Batten Disease Support and Research Association | (614) 973-6013 | i[email protected] | www.bdsra.org
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