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Treatment hope for patients with rare disorder after trial

Published on 02/05/2024

Tarekegn Hiwot, Consultant in Inherited Metabolic Disorders
Tarekegn Hiwot, Consultant in Inherited Metabolic Disorders

A new treatment, which could make a huge difference to patients with a rare hereditary disorder, has undergone a promising trial at Queen Elizabeth Hospital Birmingham (QEHB).

The experimental drug called mRNA-3927 has been tested on patients for the first time as part of a study into propionic acidaemia.

This is a serious metabolic disorder, which means the body is unable to process certain parts of proteins and fats properly. This can lead to a build-up of harmful substances the body. Without appropriate treatment, it can be fatal.

Patients with this condition must follow a specific diet, including a low protein intake and specific food for life.

Symptoms include: vomiting, lethargy, dehydration, and acid build up in the body. Liver and kidney transplant is a surgical option that can help reduce the frequency of acute metabolic episodes.

QEHB is the only adult centre in the world running this study whose initial findings have just been published.

Prof. Tarekegn Hiwot, Consultant in Inherited Metabolic Disorders at QEHB, who is leading the trial and recruited patients for the study, said: “We conducted a study of mRNA-3927 with 16 participants to find the safety, tolerability, and optimal dose.

“Our interim analysis has shown significant reduction of 70% in preventing severe metabolic crisis. The treatment was safe and well tolerated.

“In summary, this study explores a promising, first of its kind treatment for propionic acidaemia using mRNA-3927, aiming to improve patients’ health and reduce dangerous metabolic events.

“This study may also serve as a proof of concept in using mRNA treatment for other life limiting single gene genetic conditions in general.”

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