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Orphan Tech: Enzyme Replacement Therapy

Orphan Technologies is in the process of developing an enzyme replacement therapy for patients with CBS Deficiency Homocystinuria.

For the most up to date news on their enzyme replacement, please visit their website: Click here

December 22, 2017

Enzyme Replacement Therapy Ameliorates Multiple Symptoms of Murine Homocystinuria

October 16, 2017

Enzyme Replacement Therapy Prevents Loss of Bone and Fat Mass in Murine Homocystinuria.

August 16, 2017

Enzyme Replacement Prevents Neonatal Death, Liver Damage, and Osteoporosis in Murine Homocystinuria.

June 12, 2017

Engineering and Characterization of an Enzyme Replacement Therapy for Classical Homocystinuria

May 16, 2016

Journal of Clinical Investigation: Enzyme Replacement Therapy with PEGylated Cystathionine Beta Synthase Ameliorates Homocystinuria in Murine Model

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