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 Med...
Source: neuropathology blog - January 16, 2018 Category: Radiology Tags: muscle Source Type: blogs
Audentes Therapeutics Announces Dosing of First Patient in ASPIRO, a Phase 1/2 Clinical Trial of AT132 for the Treatment of X-Linked Myotubular Myopathy
Following up on the last post, Dr. Mike Lawlor sent me this in an email:"Audentes Therapeutics officially announced the dosing of the first patient for the X-linked myotubular myopathy treatment trial that we ’ve been working on over the past few years. We ’ve been very involved in the translation from dogs to humans, and will be doing the human pathology work for the trial. Here ’s a link to that press release. " (Source: neuropathology blog)
Source: neuropathology blog - October 11, 2017 Category: Radiology Tags: muscle Source Type: blogs
Lawlor featured in video about his work in myotubular myopathy
Michael Lawlor, MD, PhDMedical College of Wisconsin is highlighting the work of our colleagueDr. Michael Lawlor in the area of gene therapy for myotubular myopathy. Check out this wonderful 4-minute video! (Source: neuropathology blog)
Source: neuropathology blog - October 2, 2017 Category: Radiology Tags: muscle neuropathologists Source Type: blogs
Dr. Mike Lawlor is recognized for making critical strides in treatment of myotubular myopathy
X-linked myotubular myopathy (XLMTM) is a severe and fatal congenital myopathy for which there is currently no treatment. As part of an international group of scientists studying treatments for this disease, Dr. Mike Lawlor (the neuropathologist serving Children's Hospital of Wisconsin) was recently featured on Milwaukee's WTMJ 10 o'clock news for his part in the work. The piece is quite touching. Mike conveys a sense of urgency in finding a cure for this disease because he has gotten to know several children stricken with XLMTM, and even has pictures of many of the little tikes up on his of...
Source: neuropathology blog - April 16, 2014 Category: Pathologists Tags: muscle neuropathologists Source Type: blogs
