Mandatory Hub For Orphan-Designated Pediatric Therapy
Source: United BioSource LLC (UBC)
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.
access the Case Study!
Log In
Get unlimited access to:
Trend and Thought Leadership Articles
Case Studies & White Papers
Extensive Product Database
Members-Only Premium Content
Welcome Back! Please Log In to Continue.
X
Enter your credentials below to log in. Not yet a member of Clinical Leader? Subscribe today.
Subscribe to Clinical Leader
X
Subscribe to Clinical Leader
United BioSource LLC (UBC)
This website uses cookies to ensure you get the best experience on our website. Learn more