Search GLA Gene Variants
You can use this search tool to find out whether a specific GLA gene variant has been listed in the full prescribing information as amenable to treatment. The current list of amenable GLA gene variants can also be found in the US Prescribing Information.
Indication
Galafold® (migalastat) 123 mg capsules is indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant based on in vitro assay data.
This indication is approved under accelerated approval based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
Enter either a DNA change or protein change.
For DNA Change
Please use format c.#A>B or c.A#B for DNA changes, where 'c.' is optional; # indicates a number; A and B are letters. Examples: c.8T>C or c.T8C
For Protein Change
Protein changes can be written as either a single-letter or three-letter code. For example, p.(L3P) can be written as is or written as p.(Leu3Pro), where the ‘p.’ and ‘()’ is optional in both cases.
*Note: This is only for searching mutiple GLA gene variants on the same chromosome.
For DNA Change
Please use format c.#A>B or c.A#B for DNA changes, where 'c.' is optional; # indicates a number; A and B are letters. Examples: c.8T>C or c.T8C
For Protein Change
Protein changes can be written as either a single-letter or three-letter code. For example, p.(L3P) can be written as is or written as p.(Leu3Pro), where the ‘p.’ and ‘()’ is optional in both cases.
Enter up to three GLA gene variants:
Whether a certain amenable GLA variant in a patient with Fabry disease is disease-causing or not should be determined by the prescribing physician (in consultation with a clinical genetics professional, if needed) prior to treatment initiation. Consultation with a clinical genetics professional is strongly recommended in cases where the amenable GLA variant is of uncertain clinical significance (VUS, variant of uncertain significance) or may be benign (not causing Fabry disease).