Lucy Gavin’s son, Toby, was born eight weeks ago and the family quickly realised that something was not quite right with him.

A Mullingar mother is making an urgent appeal for access to a life-saving drug for her seven-week-old son, Toby, who has been diagnosed with a rare and complex metabolic disorder.

Moderna (MRNA), once an unknown biotech firm, quickly rose to the top of the global vaccine market during the COVID-19 ...

Titled "A Global Phase 1/2 Open-Label Dose-Optimization Study to Evaluate the Safety and Pharmacokinetics of the Biologic Treatment (mRNA-3927) in Participants with Propionic Acidemia," the research ...

As with a newborn with an organic acidemia, if the hospital is unable to offer hemodialysis, transfer of the newborn to another medical center with hemodialysis capability should be strongly ...

The endorsed benefit packages for approval include a package for acute myocardial infarction or heart attacks across the continuum of care and a package for each of these rare diseases – Maple Syrup ...

It is important to note that three of the target diseases are very rare: homocystinuria, propionic acidaemia and methylmalonic aciduria each affect only about 1 in 100,000 to 500,000 newborns.

ABSTRACT: Background: Methylmalonic aciduria (MMA) is a genetic disorder of aminoacid metabolism, due to mutations in methylmalonyl-CoA mutase, which leads to the accumulation of methylmalonic acid in ...

China's Ministry of Commerce said on Friday it will implement anti-dumping duties on propionic acid products originating from the United States for five years from July 21.

Press Releases Moderna’s Investigational Therapeutic for Methylmalonic Acidemia (mRNA-3705) Selected by U.S. Food & Drug Administration for START Pilot Program ...