Ann: ANP to present new proteomics data at WMS Congress 2021, page-45

Have you wondered about the title of the upcoming ANP presentation to the World Muscle Society? The ATL1102 treatment was in a non-ambulant cohort but it is coupled to the modulation of genetic modifiers in ambulant DMD. Why is it so?

Of course we can guess but there is no need to guess when you can simply read the scientific literature. The article Overexpression of Latent TGFβ Binding Protein 4 in Muscle Ameliorates Muscular Dystrophy through Myostatin and TGFβ published May 2016, makes the point that latent TGF-beta-binding protein 4 (LTBP4) is a genetic modifier of muscular dystrophy i.e. we are all talking about the same protein.

The article makes the point that the overexpression of LTBP4 was associated with increased muscle mass and proportionally increased strength compared to control. One would expect that an improvement to muscle mass and increased strength would prolong ambulation for boys with DMD.

But wait you say, the boys just need an increase in the level of dystrophin production and everything will be normalised. Something that struck me about the article is that the word dystrophin only appeared twice in the entire body of the article and was referenced twice in the accompanying notes. It struck me as strange because everything in the literature and the focus of drug intervention has been centred upon restoring dystrophin production. It reminds me of high school science when you learned that by holding everything constant and changing one variable you could predict the result with great accuracy. However, that rarely happens in the real world including apparently in complex biological systems.