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…

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

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…

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

Experimental Treatment for Maple Syrup Urine Disease Gets Orphan Drug Designation

According to a story from Market Screener, the international pharmaceutical company Recordati recently announced that the European Commission (EC) has recently presented the company's experimental product candidate REC 0545 with…

Continue Reading Experimental Treatment for Maple Syrup Urine Disease Gets Orphan Drug Designation
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