Social Media Ideas for Fabry Disease

Latest updates for Fabry Disease

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bioengineer.org /1 month ago

Multi-omics study identifies new drivers of organ damage in Fabry disease

A new review is reframing Fabry disease as far more than a disorder caused by the buildup of a single metabolic substance. By bringing together findings from transcriptomics, prote...

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allthatsinteresting.com /1 month ago

Inside Tomasz Nadolski’s Battle With Fabry Disease, The Disorder That Leaves Him Trapped Inside The Body Of A Child

Due to a childhood diagnosis of Fabry disease, Tomasz Nadolski hadn't yet reached puberty by the time he was 25. The post Inside Tomasz Nadolski’s Battle With Fabry Disease, The Di...

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webwire.com /2 weeks ago

FDA Approves First Therapy for Patients aged 8 years and older with Glycogen Storage Disease Type Ia

The U.S. Food and Drug Administration today issued an accelerated approval for Genglycos (pariglasgene brecaparvovec-opnr) to reduce daily cornstarch intake as an adjunct to nutrit...

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medcitynews.com /2 weeks ago

Ultragenyx Gene Therapy Becomes First FDA-Approved Treatment for Ultra-Rare Disease

Ultragenyx Pharmaceutical’s Genglycos received accelerated FDA approval for glycogen storage disease type Ia, an inherited enzyme deficiency. It’s also the first approved gene ther...

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fightaging.org /1 month ago

CMLase Enzyme Developed to Break Down CML Advanced Glycation Endproducts

Advanced glycation endproducts (AGEs) are a broad class of sugar-modified molecules that have harmful effects in the body. They are involved in generating the chronic inflammation...

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biopharmadive.com /3 weeks ago

PTC takes a chance on Sangamo’s Fabry disease gene therapy

Wall Street analysts view the deal as a risky, high-reward bet on a treatment that’s nearing a possible regulatory approval and could be superior to existing medications.

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fiercebiotech.com /5 days ago

Ionis’ ‘breakthrough’ Alexander disease approval establishes pillar of rare disease pipeline

After Jenny Pearson’s daughter Elise first had a seizure at 16 months old, an MRI came back clean—her brain “looked beautiful,” Pearson recalled to Fierce. But a follow-up scan a y...

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