The Batten Disease Support and Research Association (BDSRA) will hold its 2017 Family Conference in July, bringing together patients and families touched by Batten disease.
The on July 6–9 event will take place at the Wyndham Grand Pittsburgh Downtown. The conference joins family members, children and researchers to learn about patient care, education and the latest research.
As in previous editions, attendees will have access to presentations designed to help answer questions, connect with others, and share information. Medical care will be provided for children.
BDRSA’s tentative agenda is available here and its Facebook page includes ongoing conference updates. Registration is open until June 12, but T-shirts will only be given people who register by May 28. No registration fee is required to attend.
Conference stipends are available to families needing financial support to attend. Contact Tracy Kirby at [email protected] or 614-973-6013 for more information; Kirby is also available to answer questions regarding other matters, if necessary.
To make a reservation at the Wyndham Grand Pittsburgh Downtown Hotel, contact the hotel directly at 412-391-4600. Don’t forget to mention the BDSRA Conference to get the discounted rate of $129 per night.
Highlights from the conference’s agenda include a grandparent or extended family happy hour on Friday; “Sibs Group Research Sessions” on Friday and a trip to the nearby Carnegie Natural History Museum; a keynote research address on Saturday, followed by a children’s parade and nighttime banquet; and a Celebration of Life ceremony on Sunday.
In other news
Patients with a specific type of Batten disease now have an approved treatment,
with the U.S. Food and Drug Administration’s (FDA) recent approval of Brineura (cerliponase alfa) for use in late infantile neuronal ceroid lipofuscinosis type 2 (CLN2).
Brineura is an enzyme replacement therapy, providing CLN2 patients with a lab-produced version of TPP1 — the enzyme deficient in patients with the condition.
CLN2 is, like other types of Batten disease, caused by genetic mutations that prevent cells from effectively breaking down waste, commonly known as lysosomal storage disorders. Children with CLN2 typically develop symptoms between ages 2 and 4. Brineura is approved for the treatment of children age 3 and older.