Neurogene Initiates Natural History Study of Aspartylglucosaminuria (AGU)

oaoa | April 23, 2019

Neurogene Inc., a company founded with a mission to bring life-changing medicines to patients and families affected by rare neurological diseases, today announced that it has enrolled the first patient in a natural history study of aspartylglucosaminuria (AGU), a rare neurodegenerative lysosomal storage disorder. The goal of the study is to provide important data to support a future gene therapy clinical trial. The potential therapy is being investigated under a collaboration agreement between Neurogene and UT Southwestern Medical Center and is one of multiple sponsored agreements. Lysosomal storage disorders are a group of diseases resulting from defects in lysosomal enzyme function. Lysosomes are cellular organelles responsible for the digestion of molecules. Defective or absent lysosomal enzyme function leads to intracellular accumulation of substrate molecules, which, in turn, lead to toxicity and cell death. AGU is caused by a deficiency of the aspartylglucosaminidase (AGA) enzyme, which leads to toxic accumulation of N-acetylglucosamines and cellular dysfunction. AGU typically presents in childhood with developmental delay. With disease progression, patients experience psychomotor regression, worsening gait disturbance, behavioral and emotional issues, and worsening intellectual disability. People with AGU have a shortened lifespan, with mortality typically in the fourth decade. “This is an exciting time for people with AGU and their families,” said Kimberly Goodspeed, M.D., Assistant Professor at UT Southwestern Medical Center and lead investigator of the study.

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Your localization partner is going to do a deep-dive into your business—so start by diving deep into theirs. Are they an industry-leader with 20 years of experience or a relative newcomer? Will they speak Samoan, Uzbek, or Swahili (and all your customers’ languages)? Can they deliver on-time and on-budget—every time? Ask how many languages they support. Find out whether their translator team comprises just linguists, or linguists and subject matter experts. Explore the backgrounds of their senior management team. Be nosey. Ask questions. Cyberstalk.

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