Guest Post from Dr. Mike Lawlor: Audentes Announces Positive Interim Data from First Dose Cohort of ASPIRO, a Phase 1/2 Clinical Trial of AT132 in Patients With X-Linked Myotubular Myopathy

Dr. Mike LawlorRegular contributor Mike Lawlor, MD, PhD passed this development along from the X-linked myotubular myopathy clinical research front:Audentes Therapeutics has released an interim data update on the ASPIRO gene therapy clinical trial for X-linked myotubular myopathy.  In the 4thquarter of 2017, three patients were given a single dose of an adeno-associated virus containing the human myotubularin gene.  To quote the press release:" The early AT132 efficacy data observed in our first dose cohort of patients have exceeded our expectations, " stated Dr. Suyash Prasad, Senior Vice President and Chief Medical Officer of Audentes. " At the 12-week timepoint, Patient 1 has improved from a severely compromised baseline to achieve a CHOP-INTEND score and maximal inspiratory pressure that are approaching the ranges normally seen in healthy children.  Importantly, Patient 1 has also attained several age-appropriate developmental milestones within this time period, including head-control, rolling over and sitting unassisted.  While still early in the trial, we view these initial efficacy data as a promising indicator of the potential for AT132 to bring meaningful benefit to patients and families living with this devastating disease. "On the safety side of things, Audentes reported a total of six adverse events (AEs) reported in ASPIRO, two of which were determined to be serious adverse events (SAEs). Per the press release, “Both SAEs occurred in Pa...
Source: neuropathology blog - Category: Radiology Tags: muscle Source Type: blogs