Left ventricular hypertrophy (LVH) — a condition when the walls of the heart’s left pumping chamber (left ventricle) become thickened — accompanied by damages to peripheral nerves and hearing impairments could be signs of late-onset Fabry disease, according to a case report. “This case serves as a potent reminder…

Galafold (migalastat) is generally safe and significantly reduces disease-associated heart enlargement in Fabry disease patients carrying Galafold-amenable mutations, according to two-year data from a real-life, multicenter study in Germany. These benefits were generally similar between patients who had previously received standard enzyme replacement therapy (ERT) and those who…

Fabry disease patients treated with Galafold (migalastat) had stable kidney (renal) function for up to eight and a half years — regardless of treatment status, sex, or disease characteristics — an analysis of clinical trials found. Notably, the treatment — designed only for people with certain mutations in…

The results from a one-year extension study of the Phase 3 ATTRACT trial demonstrate the long-term safety and effectiveness of Galafold (migalastat) in people with Fabry disease with specific mutations. Importantly, the therapy’s efficacy and safety also were observed…

Galafold (migalastat) is an effective treatment for certain Japanese people with Fabry disease, with no apparent differences in response to treatment due to race, a new analysis of data from the ATTRACT study suggests. The findings were published in the Orphanet Journal of Rare Diseases in…

Nearly half of Fabry patients have amenable or responsive mutations to Galafold (migalastat) but show different responses to the therapy, according to a Swiss population study. The study found that patients with higher residual activity of the alpha-galactosidase A (GLA) enzyme — which is deficient in Fabry patients —…

Adolescents and adults with Fabry disease who live in Argentina can now be treated with Amicus Therapeutics’ oral precision medicine Galafold (migalastat). The Agency of Medicines, Food and Medical Devices (ANMAT) in Argentina has approved the use of Galafold as a monotherapy for people ages 16 or older…

Amicus Therapeutics‘ Galafold (migalastat) provides clinical benefit to patients carrying amenable Fabry disease mutations, regardless of disease severity, a Phase 3 trial shows. The findings of the study, “Efficacy of the pharmacologic chaperone migalastat in a subset of male patients with the classic phenotype of Fabry…

Galafold (migalastat) increases the activity of alpha-galactosidase A, stabilizes serum biomarkers, and improves heart function in patients with amenable Fabry disease mutations, according to clinical results. The study, “Oral Chaperone Therapy Migalastat for Treating Fabry Disease: Enzymatic Response and Serum Biomarker Changes After 1 Year,” was…

Lucerastat, an investigative oral therapy currently in late-stage clinical development at Idorsia Pharmaceuticals, successfully reduced the accumulation of a type of fat associated with disease worsening in cells from patients with Fabry disease. Researchers believe this medication is suitable for all Fabry patients regardless of their genetic background,…