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THX has granted Biocodex an exclusive global license to develop & commercialize Batten-1 for juvenile Batten disease (CLN3), along with exclusive US & Canada rights to develop & commercialize TX01 for Gaucher & Niemann-Pick type CÂ disease
As per the deal, THX will lead clinical development with financial & scientific support from Biocodex, which will…
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Sanofi has reported P-III (LEAP2MONO) trial data assessing venglustat (QD, PO) vs enzyme replacement therapy (IV, Q2W) in 43 pts (≥12yrs.) with GD3
Trial met its 1EP, improving neurological symptoms measured by modified SARA total score & RBANS at Wk. 52, & achieved 3 of 4 key 2EPs, incl. reductions in spleen & liver…
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Gaucher disease is a rare, autosomal recessive disorder that enlarges organs like the bone marrow, liver, and spleen by depositing fats (lipids) in them
In this reprise of our Disease of the Month report, we present an enlightening description of Gaucher disease with a thorough study of epidemiology, market size, disease management, potential medicines,…
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With the healthcare sector moving ahead with its technological advances, there is still an unexplored section in therapeutics that requires much more attention. Through innovations, life science companies have evolved to make breakthrough discoveries in many focus areas, but when it comes to the rare disease segment, all our endeavors seem to be bleak…

