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May 28, 2020May 28, 2020

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

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While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene ... Read more

May 26, 2020May 26, 2020

Fewer Antibodies Against PRX-102 Than Fabrazyme Likely, Trial Data Suggest

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Fabry disease patients may naturally have higher levels of neutralizing antibodies against Fabrazyme (agalsidase beta) than against Protalix BioTherapeutics‘ investigational PRX-102 (pegunigalsidase alfa), BALANCE Phase 3 trial data suggest. As ... Read more

May 25, 2020May 25, 2020

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

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European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session ... Read more

May 21, 2020May 21, 2020

Video Games Connect Chronically Ill Children Isolated at Home, Hospital

cystic fibrosis, News, syndicated

Dara Riva always had a rule that her 10-year-old son could play video games only once a week. But then the COVID-19 pandemic struck, and her perspective changed. Riva’s ... Read more

May 20, 2020

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

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Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. ... Read more

May 19, 2020May 19, 2020

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

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May 12, 2020May 12, 2020

FDA Gives Fabry Gene Therapy, FLT190, Orphan Drug Status

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The U.S. Food and Drug Administration (FDA) has designated FLT190, an investigational gene therapy for Fabry disease, an orphan drug to support and speed its development and possible review, Freeline Therapeutics, its ... Read more

May 12, 2020May 12, 2020

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

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The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people with rare diseases, their families, and caregivers. ... Read more

April 28, 2020April 28, 2020

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

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While there are few silver linings to the cloud created by COVID-19, the pandemic that has killed tens of thousands, hobbled economies worldwide and drove millions to quarantine in ... Read more

April 28, 2020April 28, 2020

A143T Mutation in GLA Gene Likely Causes Late-onset Fabry Disease, Study Suggests

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A mutation called A143T in the alpha-galactosidase A (GLA) gene is likely a cause of type 2 Fabry disease that manifests predominantly as heart problems, a study suggests. The study, “Cardiomyopathy ... Read more

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