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Research Finds RNA Therapy Factors Ameliorate SMA Phenotype

In a recent study entitled “Spinal Muscular Atrophy Phenotype Is Ameliorated Either By SMN Increase Or Modulation Of Secondary Cell Death Events With RNA Therapy (S56.006),” a team of researchers from Milan, Italy, using a stem cell model sought to develop treatment approaches based on RNA for patients with…

AveXis to Present SMA Therapy Advances at Piper Jaffray Healthcare Conference

John Carbona, CEO of AveXis, along with Dr. Brian Kaspar have been invited to present the company’s latest developments at the 26th Annual Piper Jaffray Healthcare Conference, which will take place on Tuesday, December 2. AveXis is a biopharmaceutical company currently developing gene therapy-based treatments for severe genetic and orphan diseases such as spinal muscular atrophy (SMA) in order…