Yes, at least in a certain proportion of patients.
There is always talk of an enzyme deficiency that leads to deposits in the cells and then triggers the malfunctions. In many missense variants (i.e. an amino acid is altered somewhere in the DNA on the GLA gene) there is a residual activity of the enzyme. Nevertheless, the patients are often also severely affected. Here, another mechanism comes into play: ER stress or Unfolded Protein Response (UPR).
A brief explanation: Due to the variant, the enzyme is formed, but it is partly defective. Now a kind of “quality assurance” kicks in in the cell. The faulty enzyme is “repaired”. This is a normal process in the cells, since minor errors can happen again and again. However, due to the variant, the enzyme is permanently formed incorrectly and the error correction is overloaded. This is ER stress, which can lead to malfunctions of a cell or even cause cell death.
Caperone therapy also “repairs” the wrong enzyme and relieves the cell. Here, enzyme replacement therapy would be less effective.
This mechanism has been demonstrated for some time, including for some of the controversial variants. But this explanation is not accepted by all scientists because it contradicts their own publications.
We patients are the ones who suffer; we are then prescribed no therapy or a less effective one.