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Mashup Score: 0
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: CheckRareCategories: Hem/Onc News and Journals, Latest HeadlinesTweet
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Mashup Score: 2
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: CheckRareCategories: Hem/Onc News and Journals, Latest HeadlinesTweet
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Mashup Score: 1
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: CheckRareCategories: Hem/Onc News and Journals, Latest HeadlinesTweet
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Mashup Score: 0
Robert J. Hopkin, MD, clinical geneticist, Cincinnati Children’s Hospital Medical Center, speaks on misconceptions regarding Fabry disease and opportunities for improvement in care.
Source: AJMCCategories: General Medicine Journals and Societies, Latest HeadlinesTweet
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Mashup Score: 0
A recent review highlighted the importance of managing cardiovascular risk factors in patients with Fabry disease versus the general population.
Source: AJMCCategories: Latest Headlines, PayerTweet
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Mashup Score: 0
The parameter could potentially help clinicians zero in on the cause of left ventricular hypertrophy.
Source: AJMCCategories: General Medicine Journals and Societies, Latest HeadlinesTweet
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Mashup Score: 1
Purpose of review: In this narrative review, we describe general aspects, histological alterations, treatment, and implications of Fabry disease (FD) nephropath…
Source: SAGE JournalsCategories: General Medicine Journals and Societies, Latest HeadlinesTweet
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Mashup Score: 1Understanding Fabry Disease: Perspectives from a Nephrologist - 3 year(s) ago
As a multi-systemic disorder, Fabry disease can impact the functions of various organs. As a genetic disorder, Fabry can impact the offsprings, parents, and relatives of the patient. Therefore, its effects are far-reaching.
Source: HCPLiveCategories: Latest Headlines, Rare DiseaseTweet
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Mashup Score: 0
Objective This study describes the influence of sex and disease phenotype on the occurrence of cardiac events in Fabry disease (FD). Methods Cardiac events from birth to last visit (median age 50 years) were recorded for 213 patients with FD. Patients were categorised as follows : men with classical FD (n=57), men with non-classical FD (n=26), women with classical FD (n=98) and women with…
Source: HeartCategories: Cardiologists, Latest HeadlinesTweet
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Mashup Score: 4
Introduction Recent studies showed the usefulness of globotriaosylsphingosine (lyso-Gb3) and related analogues, deacylated forms of globotriaosylceramide (Gb3), for high-risk screening, treatment monitoring and follow-up for patients with Fabry disease. Methods We evaluated Gb3, lyso-Gb3 and analogues using tandem mass spectrometry in 57 women with Fabry disease followed during a period of 15.4…
Source: Journal of Medical GeneticsCategories: General Medicine Journals and Societies, Latest HeadlinesTweet
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