ACN00177 Granted Rare Pediatric Disease Designation for Homocystinuria 
Source: Pixabay.com

ACN00177 Granted Rare Pediatric Disease Designation for Homocystinuria 

In early December, biotechnology company Aeglea BioTherapeutics, Inc. ("Aeglea") announced that its therapy ACN00177 received Rare Pediatric Disease designation from the FDA for the treatment of homocystinuria. Should this drug…

Continue Reading ACN00177 Granted Rare Pediatric Disease Designation for Homocystinuria 

A Clinical Trial Now Testing a Novel, Engineered Enzyme for Patients with Classical Homocystinuria

In a recent article appearing in BioPortfolio, Orphan Technologies announced a Phase 1/2 clinical trial involving the first Classical Homocystinuria (HCU) patients to be treated with OT-58. Twelve patients were enrolled as…

Continue Reading A Clinical Trial Now Testing a Novel, Engineered Enzyme for Patients with Classical Homocystinuria

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
Homocystinuria is More Common Than We Thought, It’s Just Not Detected Well in NBS
https://pixabay.com/en/baby-care-child-cute-hand-face-20339/

Homocystinuria is More Common Than We Thought, It’s Just Not Detected Well in NBS

Most patients diagnosed with Homocystinuria (HCU), a rare metabolic disorder, don't receive their diagnosis until later in life. Scientists were at a loss for why patients with phenylketonuria (PKU), a similar…

Continue Reading Homocystinuria is More Common Than We Thought, It’s Just Not Detected Well in NBS