Giant axonal neuropathy Archives

Tickets are on Sale Now for Rare Disease Film Festival in Boston

Post author:Jean Martell
Post published:August 8, 2019
Post category:Angelman Syndrome / Batten Disease / Giant axonal neuropathy / PACS1 Syndrome / Sanfilippo Syndrome
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Are we ready for our close-up? The second annual Selections from DISORDER: The Rare Disease Film Festival at Biotech Week Boston will be held at the Takeda Auditorium in Cambridge,…

geralt / Pixabay

Gene Replacement Research and the Hope to Cure Rare Disease

Post author:Ellen Johnson
Post published:July 1, 2019
Post category:Batten Disease / Giant axonal neuropathy
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Researchers are studying gene replacement therapy in rare diseases at UT Southwestern Medical Center. Many rare diseases are caused from a defect in a single gene, so replacing the defective…

Tickets are on Sale Now for Rare Disease Film Festival in Boston

Gene Replacement Research and the Hope to Cure Rare Disease

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