July/August Issue of the Illuminator

August 2016 Newsletter_Page_1The July/August 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 grant awards, family resources, board elections and more.
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June Issue of the Illuminator

June 2016 Newsletter_Page_1The June 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 virtual 5K results, conference news, fundraisers and more.
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May Issue of the Illuminator

May 2016 Newsletter_Page_1The May 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 conference registration, Batten Awareness Weekend, fundraisers and more.
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2016 BDSRA Annual Family Conference

BDSRA 2016 Conference LogoThe hotel reservation link is now closed.  If you have not made your room reservations, please contact the hotel Reservation Coordinator, Charlotte Tourkakis  ctourkakis@renaissancestlouisap.com  or 314.890.3151. Please note that room reservations are based on availability. Please contact Sara Aldrich at 518.930.2673 or sarab@sswmeetings.com for conference registration. To see the tentative conference agenda click here 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. and under the umbrella of “Family Support Services” financial help may be available. To see if family support funds might be available in local regions through the DD council, visit http://www.nasddds.org/state-agencies/ If you need any assistance locating your county office or need additional information about this year’s conference, please do not hesitate to contact Becky Hetteberg, BDSRA Director of Family Support and Education at 1-800-448-4570 ext.
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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.
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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.
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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. 
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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.
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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
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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.
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