Human RNAi Therapy Jumps Hurdle

The authors identified only one clear adverse event in the human study: a moderate infusion-related reaction in the patient who received 0.5 mg/kg of ALN-TTR02. Mild-to-moderate infusion-related reactions occurred in 20.8% of patients receiving ALN-TTR01 and 7.7% of patients receiving ALN-TTR02.

Dr. Suhr expressed hope that it may be possible to bring the RNAi therapy to market in 2017 or 2018.

Dr. Pullen is a medical writer based in the Chicago area.

Reference

Coelho T, Adams D, Silva A, et al. Safety and efficacy of RNAi therapy for transthyretin amyloidosis. N Engl J Med. 2013;369:819-829.

Although amyloidosis is not a rheumatic disease, rheumatologists may be the first to diagnose this life-threatening condition

CHICAGO—Caryn A. Libbey, MD, clinical associate professor of medicine at Boston University School of Medicine, described the evolving in our understanding of amyloid at the ACR’s State-of-the-Art Clinical Symposium in April. Amyloidosis is a rare disease that is often underdiagnosed and undertreated. “Even though this disease has been around for 150 years, I still consider it…

SAN DIEGO—Treatments do exist that can improve the prospects of a patient with the rare disease amyloidosis, but only if clinicians keep the disease in mind and treat the patient before it’s too late, an expert said at the 2017 ACR/ARHP Annual Meeting this past November. He also discussed research that may be close to…

SAN FRANCISCO—Treatment for transthyretin-related amyloidosis (ATTR) is generating more interest from academic researchers and the pharmaceutical industry, with encouraging early results using a multi-pronged therapeutic approach, a researcher said at a review course held before the 2015 ACR/ARHP Annual Meeting. Amyloidoses are a rare and potentially deadly family of diseases in which misfolded protein builds…

Reference

What Rheumatologists Need to Know about Identifying Amyloidosis

Amyloidosis Is Often Underdiagnosed, Undertreated

An Amylodosis Warning & New Drug Hope

2015 ACR/ARHP Annual Meeting: Treatments for Transthyretin-Related Amyloidosis Generate Interest from Researchers, Pharmaceuticals

Reference

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