Shortly after Laura Colby was born, she was diagnosed with a rare metabolic disorder known as maple syrup urine disease (MSUD). Normally, our body has an enzyme complex that breaks…
We know that it can be difficult to receive the correct diagnosis for a rare disease. Sometimes, you have to be your own advocate. This means learning everything you can,…
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…
According to a story from Medical Xpress, a recent study that draws on 30 years of maple syrup urine disease patient data has concluded that while significant improvements have been…
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…
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…
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