Newsletter
Newsletter

Two Siblings with SMA Receive Gene Therapy

by | Mar 12, 2021

   Emily Holtvluwer discusses the gene therapy her two children with spinal muscular atrophy (SMA) have received.   As Ms. Hotvluwar explains, her oldest child was diagnosed several months after symptoms began to appear while her second child was diagnosed in...

Gene Therapy and Sanfilippo Syndrome Type A

by | Mar 11, 2021

  Michael Hocquemiller, PhD, and Samantha Parker of Lysogene talk about the latest developments in the company’s gene therapy, LYS-SAF302, to treat Sanfilippo syndrome type A. Sanfilippo syndrome type A (MPS IIIA) is a progressive, life-threatening, and rare...

SMA Type 2: A Diagnostic Journey

by | Mar 11, 2021

  Regina Phillips, mother of a young child diagnosed with spinal muscular atrophy (SMA), talks about the 4 month journey to get properly diagnosed with this rare neuromuscular disorder.  As Ms. Phillips explains, her son, Shane, was born looking very happy and...

Two Tips to Better Manage Your Pompe Patient

by | Mar 10, 2021

  Melanie McKay, mother of a young boy with infantile-onset Pompe disease talks about the importance of having a clinician listen to the caregiver of the child. Pompe disease is a rare inherited lysosomal storage disorder due to low levels of acid...