In Europe, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare, life-threatening, or chronically debilitating conditions. For the European Union (EU), a “rare”…
In a late January 2022 news release, gene therapy company Taysha Gene Therapies, Inc. ("Taysha") shared that trial data was now available on TSHA-120. The therapy, designed to treat giant…
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,…
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…
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