Opfolda (miglustat) is a medicine taken with another medicine for certain people with late-onset Pompe disease. Pompe disease is a rare genetic disorder that is caused by an abnormal gene for a ...
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Bayer backs AskBio gene therapy push in Pompe disease: Early trial update and investor takeaways
Bayer AG (BAYRY) announced an update on their ongoing clinical study. Study Overview The PROGRESS-GT LOPD trial is an early-stage clinical study sponsored by AskBio, with Bayer as a collaborator, to ...
Amicus Therapeutics has released a post-hoc analysis from the PROPEL study, published in Muscle and Nerve, which evaluated the efficacy of cipaglucosidase alfa-atga combined with miglustat (cipa+mig) ...
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