Batten Awareness Weekend Virtual 5K

Batten Awareness Weekend Virtual 5K  We are very excited to announce the BDSRA Batten Awareness Weekend Virtual 5K! Each year, during the first weekend in June, the Batten Disease Support and Research Association celebrates Batten Awareness Weekend to promote the need for research and support for families. The race will take place during awareness weekend, June 4th and 5th.  A virtual 5K is just like any other 5K, except there is no official race location.  That also means no early wake up times and no traveling.  You can choose when, where and how you complete the 5K. Once you are done, log back in and enter your time.  The top finishers in individual and team categories and the top fundraisers will be acknowledged at the annual BDSRA family conference in July of this year, as well as in the newsletter and on our website.  There are also options for those who want to join in the weekend event but don’t want to run a race.
(Read More)

2016 BDSRA Annual Family Conference

BDSRA 2016 Conference LogoHotel reservations are now being accepted by the Renaissance St. Louis Airport Hotel, site of this year’s Batten family conference, July 14-17.  To reserve your room, please visit http://cwp.marriott.com/stlsa/bdsra2016/ . If you would like to make your reservations by phone, please call 1.800.468.3571 and mention “BDSRA Batten disease” to get the discounted rate of $105 per night.   The conference room block dates are July 13, 14, 15 and 16. If you need additional dates or would like to book a suite, please contact the hotel Reservation Coordinator, Charlotte Tourkakis  ctourkakis@renaissancestlouisap.com  or 314.890.3151. Please note that additional dates are based on room availability.  Reservations must be made by June 21, 2016. Online conference registration is available at: http://bit.ly/1Qyu5xv For families seeking financial support to attend conference, one source for potential support is contacting the local Developmental Disability Council. There are Developmental Disability (DD) Planning Councils that serve each state and territory in the U.S.
(Read More)

March Issue of the Illuminator

March 2016 Newsletter_Page_1The March issue of the BDSRA newsletter The Illuminator is now available.  We will be sending a monthly e-newsletter so that we can share more information with you more often.  This replaces our quarterly format. Click here to read about CLN2 ERT results, CLN3 update, Rare Disease week and more.
(Read More)

Call on Congress to Pass the OPEN ACT

open act 1Last year, the OPEN ACT, legislation that could double the number of treatments for rare disease, was left out of the draft of the House Cures bill.  But policy makers had a change of heart after hundreds of patient advocates flooded the phone lines and asked Congress to put OPEN back in.  Now, the OPEN ACT has again been left out again by the Senate, despite the support of over 160 national patient organizations!  We need your help once again to get the OPEN ACT back in action.  Please take a few moments to call Senators Alexander (R-TN) and Murray (D-WA) TODAY to ensure they help rare disease patients!  Your call will make the difference. To contact Sen. Lamar Alexander, dial: (202) 224-4944 To contact Sen. Patty Murray, dial: (202) 224-2621 Script for Senators Alexander & Murray: My name is [NAME]. I’m calling today as a rare disease patient advocate [Briefly explain your disease, 2-3 sentences] We urge you to support the OPEN ACT, S.
(Read More)

BioMarin Announces Positive Data for Treatment of CLN2

BioMarin Announces Positive Data for Treatment of CLN2SAN RAFAEL, Calif., March 02, 2016 (GLOBE NEWSWIRE) — BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced positive 48-week results from its Phase 1/2 pivotal study for cerliponase alfa, a recombinant human tripeptidyl peptidase 1 (rhTPP1) to treat children with CLN2 disease, a form of Batten disease.  CLN2 disease is a rapidly progressing, fatal neurodegenerative disease with no approved treatments, where the majority of affected children lose their ability to walk and talk by approximately six years of age.  The average rate of clinical decline for motor and language function in patients receiving cerliponase alfa treatment — the primary efficacy endpoint — was approximately 80% less than the expected rate of decline in the untreated population, preserving essential function in the majority of treated patients (p <0.0001).  Treatment with 300 mg cerliponase alfa administered via intracerebroventricular (ICV) infusion every other week was generally safe and well-tolerated in 24 patients and resulted in disease stabilization in 65% (15 of 23) of patients treated over a 48-week period, based on the Hamburg Motor + Language CLN2 rating. 
(Read More)

February Issue of the Illuminator

Feb 2016 Newsletter_Page_1The February issue of the BDSRA newsletter The Illuminator is now available.  We will be sending a monthly e-newsletter so that we can share more information with you more often.  This replaces our quarterly format. Click here to read about researcher updates, membership, conference and more.
(Read More)

2016 Annual Membership

Membership Website 2Show Your Strength: February is membership month
for BDSRA. To join or renew your 2016 annual membership
go to Become A Member Why Join BDSRA?  Because membership is a vital link to others
facing Batten disease, and membership strength is a leading indicator
to policy makers and industry that Batten disease deserves
attention, support and funding. For questions email tkirby@bdsra.org
(Read More)

January Issue of The Illuminator

Jan 2016 Newsletter Edits_Page_1The January issue of the BDSRA newsletter The Illuminator is now available.  We will be sending a monthly e-newsletter so that we can share more information with you more often.  This replaces our quarterly format. Click here to read about research, board elections, fundraisers and more.
(Read More)

BDSRA Annual Family Conference

BDSRA 2016 Conference LogoThe 2016 family conference will be in St. Louis, Missouri, July 14-17 at the Renaissance St. Louis Airport Hotel. Be sure to visit our 2016 BDSRA Conference page on Facebook and “like” the page to stay connected. We are excited to share the new conference logo and to be heading to the Show Me State for our BDSRA Family Conference. Online registration and hotel details will be available mid-February. Check back here often for updates and registration information. We look forward to seeing everyone in July.
(Read More)