Strimvelis Gene Therapy for ADA-SCID Connected to Leukemia Case

  While gene therapy offers the promise of addressing and treating many genetic disorders, creating a better quality of life for many people, there are also ethical issues associated with…

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ACR Convergence 2020: Data Available on Octagam 10% for Dermatomyositis
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ACR Convergence 2020: Data Available on Octagam 10% for Dermatomyositis

  This year, the American College of Rheumatology (ACR) held their Convergence 2020 meeting virtually from November 5-9, with additional poster presentations on November 10 and 21. According to a…

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Possible Treatment for Pompe Disease to be Evaluated Under Priority Review
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Possible Treatment for Pompe Disease to be Evaluated Under Priority Review

According to a press release from the biopharmaceutical company Sanofi, the Biologics License Application (BLA) for its drug avalglucosidase alfa has been accepted by the US Food and Drug Administration…

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Update On Newborn Screening Efforts for Duchenne Muscular Dystrophy
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Update On Newborn Screening Efforts for Duchenne Muscular Dystrophy

Parent Project Muscular Dystrophy (PPMD) is a nonprofit that focuses on fighting for a cure, better resources, and more awareness for Duchenne muscular dystrophy (DMD). More than a year ago…

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Charcot-Marie-Tooth Disease Patients Report Declines During COVID-19 Lockdown
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Charcot-Marie-Tooth Disease Patients Report Declines During COVID-19 Lockdown

According to a story from Charcot-Marie-Tooth News, patients living with Charcot-Marie-Tooth disease have reported in a recent survey that their symptoms of pain and decline in mobility increased during the…

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