Provide Advice Regarding Stem Cells And Gene Therapy For One With Genetic Mutation Of Adipocyte Triglyceride Lipase Deficiency And Has Cardiac Involvement

Posted on Fri, 29 Jun 2018

Medically reviewed by Ask A Doctor - 24x7 Medical Review Team

Fri, 29 Jun 2018 Answered on

Wed, 9 Feb 2022 Last reviewed on

Question : Have a 50 year old male patient with diagnosed genetic mutation of adipocyte triglyceride lipase deficiency and has cardiac involvement and is seeking help of any type. Asked me about stem cells and gene therapy. Thoughts?

Answered by Dr. Mirjeta Guni (7 hours later)

Brief Answer:
About glybera;

Detailed Answer:

Hello,

Yes, you may suggest to your patient gene therapy.
The gene-therapy product Glybera (alipogene tiparvovec), approved by the European Medicines Agency in 2012 for patient with lipoproteine lipase deficiency who have a condition that can cause life threatening (pancreatitis, myocardial infarction ) diseases can be effective in reducing 40% fasting tryglicerides reducing the risk for severe complications.

In your living area, I do not know if it is available but he should get interested at pharmaceutical companies.

Best wishes,
Dr.Mirjeta

Above answer was peer-reviewed by : Dr. Nagamani Ng