Most children with Fabry disease, particularly those with the severe, classic form, have lower than normal skeletal muscle mass that affects the lower limbs more than the upper limbs, a study in China shows. Skeletal muscles are responsible for voluntary movements and play a vital role in everyday…
4D Molecular Therapeutics (4DMT) has reached an agreement with the U.S. Food and Drug Administration (FDA) to lift the hold on a U.S.-based clinical trial testing 4D-310, an investigational gene therapy for Fabry disease. The hold was based on safety data from the company’s INGLAXA Phase 1/2 trials.
About a third of Fabry disease patients were found to have low levels of antibodies against a substance called polyethylene glycol, or PEG, that’s present in Elfabrio (pegunigalsidase-alfa), an enzyme replacement therapy approved in the U.S. earlier this year as a treatment for the genetic disorder. However, these…
A review study revealed that those with Fabry disease have a high prevalence of a wide range of sleep problems, with excessive daytime sleepiness being the most common. Reviewed studies also commonly reported insomnia, unrefreshing sleep, periodic limb movements, and REM sleep behavior disorder, or physically acting out dreams…
Messenger RNA (mRNA) treatment restored levels of Gal A enzyme activity in heart cells derived from two Fabry disease patients with severe heart involvement, a study showed. The mRNA therapeutic delivered the correct information contained in the GLA gene to produce a fully functional Gal A enzyme, which is…
A team of expert clinicians and patient advocates has created a new set of recommendations to guide the use of Galafold (migalastat) in people with Fabry disease, highlighting the importance of centering patients’ preferences in treatment decisions. “We hope that this publication will lead to the provision of…
The duration and rate of Fabrazyme (agalsidase beta) infusion can be safely changed in people with Fabry disease, independently of their body weight, if appropriate care is taken, according to a new study from Japan. Specifically, an infusion time shorter than 90 minutes or an infusion rate higher…
A man diagnosed in his 60s with late-onset Fabry disease had an “uneventful” recovery after receiving both a heart and a kidney transplant in surgeries seven years apart, according to a new case report. “Heart and kidney transplants can play a major role in patients with Fabry disease who develop…
The National Institute for Health and Care Excellence (NICE) in England has recommended that Elfabrio (pegunigalsidase alfa) be covered by the country’s national health service (NHS) and provided at low or no cost to adults with Fabry disease. About 1,150 people in England are estimated to have Fabry,…
People with Fabry disease who have burning limb pain or depression are more likely to report poor quality of life, a new study shows. Addressing these issues may help them, researchers said in “Screening for health-related quality of life and its determinants in Fabry disease: A…
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