• Mashup Score: 3
    FDA Approval of ELFABRIO - 12 month(s) ago

    – PEGylated enzyme replacement therapy designed to provide long half-life*. BOSTON and CARMIEL, Israel, May 10, 2023 – Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people affected by rare diseases, and Protalix BioTherapeutics, Inc. (NYSE American:PLX), a biopharmaceutical company focused on the development and…

    Source: chiesirarediseases.com
    Categories: Cardiologists, Latest Headlines
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    • Profile photo of MasriAhmadMD
      MasriAhmadMD

      Elfabrio (great name!) is now approved by @US_FDA See a refresher below on Fabry and new treatments. Still awaiting the label to be published Congratulations to the #Fabry & #raredisease community for getting another option for ERT #CardioTwitter https://t.co/MvF21ne5X9 https://t.co/DEGiBtGaVQ

  • Mashup Score: 0
    Effect of Migalastat on cArdiac Involvement in FabRry Disease: MAIORA study - 1 year(s) ago

    Background A small but significant reduction in left ventricular (LV) mass after 18 months of migalastat treatment has been reported in Fabry disease (FD). This study aimed to assess the effect of migalastat on FD cardiac involvement, combining LV morphology and tissue characterisation by cardiac magnetic resonance (CMR) with cardiopulmonary exercise testing (CPET). Methods Sixteen…

    Source: Journal of Medical Genetics
    Categories: General Medicine Journals and Societies, Latest Headlines
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    • Profile photo of JMG_BMJ
      JMG_BMJ

      #Fabry Disease - New treatment trial (MAIORA study) Migalastat stabilised cardiac mass & trend towards an ⬆️ exercise tolerance 👀 https://t.co/NeO4Or0FTN #Genetics #PersonalisedMedicine #CardioTwitter @MPSSocietyUK @FabryDisease1 @fabryalerts @GuazziMarco @lara89eleven https://t.co/1nqW31iP1z

  • Mashup Score: 0
    Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease - Gene Therapy - 1 year(s) ago

    Fabry disease is an X-linked lysosomal storage disorder caused by loss of alpha-galactosidase A (α-Gal A) activity and is characterized by progressive accumulation of glycosphingolipids in multiple cells and tissues. FLT190, an investigational gene therapy, is currently being evaluated in a Phase 1/2 clinical trial in patients with Fabry disease (NCT04040049). FLT190 consists of a potent,…

    Source: Nature
    Categories: Cardiologists, Latest Headlines
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      MasriAhmadMD

      More on gene therapy but this time for #Fabry Preclinical evaluation of FLT190 - doing everything it is supposed to do https://t.co/RSdz7djkLy MARVEL Phase 1/2 is ongoing in Europe sponsored by @FreelineLife #CardioTwitter https://t.co/0XlwBknql2

  • Mashup Score: 21
    Echocardiography in Anderson-Fabry Disease - 2 year(s) ago

    Echocardiography is the most common diagnostic tool to screen for Fabry cardiomyopathy as it is fast, non-invasive, low-cost, widely available, easily applicable and reproducible. Echocardiography is the first-line investigation, being useful in all the stages of the disease: (1) in gene-positive patients, to unveil signs of early cardiac involvement and allowing timely treatment;…

    Source: IMR Press
    Categories: Cardiologists, Latest Headlines
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    • Profile photo of purviparwani
      purviparwani

      RT @FGraziani_Grace: Interested in #echofirst in #Fabry disease? Enjoy our #review on the topic! 📚🫀 ⬇️⬇️⬇️https://t.co/u40RW5JEG0 @dr_benoy…

  • Mashup Score: 3
    Echocardiography in Anderson-Fabry Disease - 2 year(s) ago

    Echocardiography is the most common diagnostic tool to screen for Fabry cardiomyopathy as it is fast, non-invasive, low-cost, widely available, easily applicable and reproducible. Echocardiography is the first-line investigation, being useful in all the stages of the disease: (1) in gene-positive patients, to unveil signs of early cardiac involvement and allowing timely treatment;…

    Source: IMR Press
    Categories: Cardiologists, Latest Headlines
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    • Profile photo of KemalogluOz
      KemalogluOz

      RT @FGraziani_Grace: Interested in #echofirst in #Fabry disease? Enjoy our #review on the topic! 📚🫀 ⬇️⬇️⬇️https://t.co/u40RW5JEG0 @dr_benoy…

  • Mashup Score: 0
    Promising Data from Investigational Gene Therapy for Fabry Disease - 2 year(s) ago

    Dr. Jerry Vockley, of UPMC Children’s Hospital, discusses the results of the phase 1/2 trial  testing a new gene therapy, 4D-310, in Fabry disease patients.

    Source: CheckRare
    Categories: Hem/Onc News and Journals, Latest Headlines
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      CheckRare

      Jerry Vockley, MD, PhD, Head of the Division of Medical Genetics at UPMC Children’s Hospital of Pittsburgh, discusses the phase 1/2 trial of the gene therapy, 4D-310, in Fabry disease. @ChildrensPgh @FabryDisease1 @FabryOrg @TUAFD1 #Fabry https://t.co/oL4CkLLQtL

  • Mashup Score: 1
    Promising Data from Investigational Gene Therapy for Fabry Disease - 2 year(s) ago

    Dr. Jerry Vockley, of UPMC Children’s Hospital, discusses the results of the phase 1/2 trial  testing a new gene therapy, 4D-310, in Fabry disease patients.

    Source: CheckRare
    Categories: Hem/Onc News and Journals, Latest Headlines
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    • Profile photo of CheckRare
      CheckRare

      Jerry Vockley, MD, PhD, Head of the Division of Medical Genetics at UPMC Children’s Hospital of Pittsburgh, discusses the phase 1/2 trial of the gene therapy, 4D-310, in Fabry disease. @ChildrensPgh @FabryDisease1 @FabryOrg @TUAFD1 #Fabry https://t.co/oL4CkLLQtL

  • Mashup Score: 2
    Promising Data from Investigational Gene Therapy for Fabry Disease - 2 year(s) ago

    Dr. Jerry Vockley, of UPMC Children’s Hospital, discusses the results of the phase 1/2 trial  testing a new gene therapy, 4D-310, in Fabry disease patients.

    Source: CheckRare
    Categories: Hem/Onc News and Journals, Latest Headlines
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    • Profile photo of CheckRare
      CheckRare

      Jerry Vockley, MD, PhD, Head of the Division of Medical Genetics at UPMC Children’s Hospital of Pittsburgh, discusses the phase 1/2 trial of the gene therapy, 4D-310, in Fabry disease. @ChildrensPgh @FabryDisease1 @FabryOrg @TUAFD1 #Fabry https://t.co/oL4CkLLQtL

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