Investigative drug approved
The Canadian charity CureSPG50 is pleased to announce that the U.S Food and Drug Administration (FDA) has awarded Rare Paediatric Disease Designation (RPDD) to Melpida an investigation treatment for SPG50.
CureSPG50 is developing Melpida, which seeks to slow the progression of the inherited neurodegenerative condition Spastic Paraplegia Type 50 also known as SPG50. SPG50 causes children affected by this terrible disease to become completely paralyzed with limited brain function.
“We are extremely pleased to have obtained Rare Paediatric Disease Designation (RPDD),” said Terry Pirovolakis, who along with his wife Georgia started CureSPG50 after their son Michael was diagnosed with the rare genetic disease as an infant.
The clinical study will compare Melpida’s effectiveness in pediatric children, to the natural progression of SPG50 as documented in previous groups of patients. CureSPG50 is still in talks to determine who will be the lead investigation center for the trial.
Read the full article.
CureSPG50 Receives FDA Rare Paediatric Disease Designation Status For The Treatment of SPG50
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