Newer disease-modifying therapies for hereditary amyloidosis can improve symptoms and enhance the quality of life for patients with hATTR.
Clinical Professor, Department of Neurology, University of Pennsylvania School of Medicine; Director, Clinical Neurophysiology and Outpatient Services, Clinical Practices of the University of Pennsylvania , Philadelphia, Pennsylvania Hello. My name is Sami Khella. Thank you for joining me in this session on the treatment of hereditary transthyretin polyneuropathy and cardiomyopathy. The treatment can be broken up into symptom management and disease modification.
This drug can also be used in the very early state when the patient manifests the disease itself. However, the gold-standard therapies are the silencers of TTR. There are currently three FDA-approved drugs. These includeThe choice among these is really patient preference and perhaps patient side effects, as there are no head-to-head trials showing one of these drugs to be more effective than the other.
The small publications so far have shown that there are no serious side effects from these drugs, but whether there is benefit is not entirely clear. For patients who have central nervous system disease, there are no FDA-approved therapies at this time. And those patients may be more frequently seen, as the patients with the cardiomyopathy polyneuropathy hopefully live longer.has been shown to possibly be effective.
Whether measuring prealbumin is useful is not entirely clear. It may be helpful to measure prealbumin if you're looking for efficacy of the drug in terms of TTR suppression. The goal of the therapy is prevention. And if you have improvement in the symptoms rather than simply prevention of progression, that may be an added bonus. Excitingly, on the horizon, is CRISPR-Cas9, but those trials are ongoing at this time.
Any views expressed above are the author's own and do not necessarily reflect the views of WebMD or Medscape.
Disease Modifying Therapy DMT - Disease Modifying Therapy Cardiomyopathy Orthostatic Hypotension Pennsylvania Adverse Effects Side Effects Biomarker Biological Marker Multi-Biomarker Disease Activity MBDA Multibiomarker Disease Activity Hypotension Quality Of Life QOL Health Related Quality Of Life Health-Related Quality Of Life HRQOL Transplantation Of The Liver
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