India Has Introduced a New Rare Disease Policy Draft, But it Doesn’t Benefit all Rare Patients
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India Has Introduced a New Rare Disease Policy Draft, But it Doesn’t Benefit all Rare Patients

India first drafted a national rare disease policy back in 2017. Unfortunately, it was never enacted due to budgeting and implementation issues. This draft included suggestions such as a corpus…

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Attention Tyrosinemia Community Families: Come Together for Event in Late September

September is Tyrosinemia Awareness Month! The good people of the Network Of  Tyrosinemia Advocates (N.O.T.A.) have organized a family weekend later this month aimed to bring the Tyrosinemia community together. Let's…

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Yeast May be the Key to Uncovering Treatments for Homocystinuria and Other Metabolic Conditions

There is a great unmet medical need for patients living with metabolic disorders. There are few approved treatments for these conditions and many are fatal. These diseases include phenylketonuria, homocystinuria, tyrosinemia, and maple syrup…

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Scientists are Using Pre-Natal Genetic Editing to Treat Hereditary Tyrosinemia Type 1 in Mice

Researchers have succeeded in editing genes to prevent a metabolic disorder called hereditary tyrosinemia type 1 (HT1) in animals while they are still in the womb. The team, who are…

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This Gene Editing Approach Could Help Treat Genetically Linked Rare Diseases

According to a story from medicalxpress.com, a team of scientists at the University of Massachusetts Medical School have successfully developed a strategy for editing genes that could be used to…

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Cycle Pharmaceuticals Partners With US-Firm To Utilize 3D Printing Technology For Rare Disease Treatment Development
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Cycle Pharmaceuticals Partners With US-Firm To Utilize 3D Printing Technology For Rare Disease Treatment Development

Cycle Pharmaceuticals shook hands with US-based firm, Aprecia Pharmaceuticals as they agreed to develop and commercialize treatments for rare diseases using the advanced technology of 3D printing, recently reported Cambridge…

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You Can Dance the Night Away to Raise Money for Children’s Miracle Network Hospitals!
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You Can Dance the Night Away to Raise Money for Children’s Miracle Network Hospitals!

If dance is the universal language, then what does NC State’s annual Dance Marathon have to say--especially to type 1 tyrosinemia (TYR)? A lot, it turns out. The numbers seem…

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