On ClinicalTrials.gov you will now see that the ACE-031 study is terminated.
While this news is frustrating, it is not unusual in drug development. We all wish it would be a straight path through each phase of the clinical trial process to approval, but that is often not the case. Drug development often has stops and starts, when a SAE (serious adverse event) occurs or questions about dosing arise. This results in days and weeks of discussions about the cause of a…Continue
first conference in Merida, Yucatan, Mexico. This is a major step for UPA! and will be focused on promoting early and accurate diagnosis and standards of care. Their intention is to identify healthcare professionals with a willingness to specialize in interdisciplinary care for Duchenne muscular dystrophy. May 14 will include families and patients as a first step to…Continue
Added by Pat Furlong on April 20, 2011 at 1:00pm — No Comments
April 7-9, 2011
The flight from LAX to Guangzhou was 15 hours or so. It was long and felt a little frightening, heading to a country vastly different from my own. My very limited knowledge of China has been acquired from tidbits on the news. I was aware that for the last 20 years or more, China has practiced family planning as a basic policy. This family planning policy advocates each couple to have one child in accordance with the family planning policy.…Continue
Added by Pat Furlong on April 12, 2011 at 9:26am — No Comments
I’ve just spent two weeks in a row on the road at different meetings—first the Health Research Alliance (HRA) meeting in New York and then the Muscular Dystrophy Association meeting in Las Vegas.
HRA is an organization made up of most of the major non-profit funders of health research -- the members meetings are designed to keep us up to date on the latest trends in research and funding and to allow us to learn from one another. I chair the HRA Working Group on “Funders of…Continue
This is one of the most difficult blogs I have ever written, but I wanted to make sure the community heard it from us first and has accurate information.
Lee Sweeney has been diagnosed with throat cancer. The diagnosis came on an ordinary day. Lee and I were on the phone on a regular ‘catch up’ call. Lee had spent the previous two weeks in Dallas. His father had a stroke and Lee (typical) was caring for his dad and holding his mom and sisters together. There was fatigue in…Continue
The Story of Ataluren, or in simple terms: Looking beyond the p-value
I recently participated in a “DAY OF DIALOGUE, ” in an effort to explore the development of ataluren, the story of the science of post transcription , or the possibility and probability of interfering with mRNA in order to affect the course of a disease. Thankfully, they started with Duchenne.
Founded in 1998, PTC has focused on developing ataluren as well as other…Continue