Case Study

Mandatory Hub For Orphan-Designated Pediatric Therapy

pediatric patient

UBC engaged with a client whose product has Orphan Designation, and is Indicated for the treatment of Spinal Muscular Atrophy (SMA)-- SMA is caused by a mutation in the SMN1 gene, which encodes for a protein that motor neurons, need in order to survive. The mutation prevents this gene from producing any functional SMN protein which can lead to death or the need for mechanical ventilation by two years of age. Our client’s product is considered a life-saving therapy for patients and their families; however, it comes with a very high cost.

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