<ul>
 <li>blood-brain barrier</li>
 <li>substrate reduction therapy</li>
  <li>miglustat</li>
  <li>venglustat

</li>
</ul>
<p><br></p>

blood-brain barrier substrate reduction therapy miglustat venglustat

Emerging Treatments for TSD and Sd

Faculty experts, Jeanine Jarnes, PharmD, and Chester Whitley, MD, PhD delve into the rare and difficult to diagnose GM2-gangliosidoses Tays-Sachs and Sandhoff diseases. Following a discussion of the etiology and pathophysiology of these diseases, the faculty use real-life cases to illustrate the various phenotypes of these conditions, as well as key presenting signs and symptoms. Diagnostic approaches are discussed, alongside current standards of care, including supportive and palliative treatment options. Drs. Jarnes and Whitley describe key therapeutic approaches under investigation to impro

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Emerging Treatments for TSD and Sd

Emerging Treatments for TSD and Sd