Sponsor or institution

Clinical trials and research at Biostrategies, Lc

• 3NIH research projects

NIH-funded research (3)

Early-stage science funded by the US National Institutes of Health. Most of these projects don't enroll patients, but they show what is being worked on.

• Treating GM1 brain disease with a systemically delivered enzyme plus gene therapy

  A new therapy for GM1 gangliosidosis aims to deliver a corrective enzyme to the brain and body using a one-time AAV-based approach to help children with GM1.

  NIH research Biostrategies, Lc · State University, United States · NIH-11627834

• Gene therapy to deliver a replacement enzyme for Sanfilippo A syndrome

  This study is testing a new gene therapy for people with Sanfilippo A syndrome that aims to deliver a helpful enzyme to the brain and other parts of the body without needing any invasive procedures, with the hope of easing symptoms and improving daily life.

  NIH research Biostrategies, Lc · State University, United States · NIH-10930137

• New therapy combining gene transfer and enhanced enzyme delivery for treating Morquio A Syndrome in children

  This study is testing a new treatment for kids with Morquio A Syndrome that uses a special method to deliver an important enzyme, hoping to help them feel better and improve their breathing, heart health, and movement.

  NIH research Biostrategies, Lc · State University, United States · NIH-10821924

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