Without Endings There Are No Beginnings…

It’s New Year’s Day 2015 and I think Janus, the two-headed Roman God who looks forward and backwards at the same time, is a fitting representation of my life right now. I need to tell you all that I have had to make a difficult decision this year—at the end of this month I will be winding up my time at PPMD while simultaneously starting mid-month with the Myotonic Dystrophy Foundation (MDF) as its first research director.

Several key factors have motivated this decision. First, many of you know that my eight-year-old (almost nine year old) son Pierce is a delightful, creative little nut who never fails to surprise me with his own unique view of the world. You may also know that he is on the autism spectrum—kind of mild, but kind of not. And as you all know, raising a child with a disability is fraught with second-guessing and worry. As my son approaches his pre-teen years, we know that things are going to get a lot more complicated for him and much of the shielding his father and I have provided will need to be replaced by his own hard-earned coping skills. I need to be more available to my son as he navigates these waters.

At the same time, my husband Scott has recently become the Executive Director of the Pima Air and Space Museum here in Tucson (very proud of him). This is a position for which he is well qualified and I know he will excel, but it means that we are now permanently on the West coast. It also means that Scott, who has been an amazing single dad much of the time while I have traveled for PPMD, now needs me here to help juggle school schedules and pets, etc. as he has late board meetings and breakfasts with donors.

Moving to MDF will not mean an easier job by any means, but it will mean less travel as that organization’s headquarters are on the West coast and the myotonic dystrophy field is in a different place than Duchenne. It will also reconnect me with a community that I knew well through my time at the Muscular Dystrophy Association and, most importantly, allow me to continue to work in the neuromuscular space while balancing family obligations.

So what can I say about my time at PPMD?

First, I’m most proud of establishing the Duchenne Drug Development Round Table—an informal neutral forum that allows all the companies working in the Duchenne drug development space to interact freely around common issues. This group has met five times now, has a mailing list of 75 people, and has tackled such topics as phase IV surveillance studies, recruitment competition, and the use of natural history data to interpret trial results. I am also happy to have reorganized PPMD’s Scientific Advisory Committee, and moved PPMD’s grant review and management process out of Word folders and into a professionally managed database.

During my time at PPMD we have also funded Jerry Mendell’s successful follistatin gene therapy trial, the pilot study at Cedars Sinai that led to the Wyeth phase III study of Tadalafil, and the eplerenone cardiac study, whose positive results were just announced. As I leave, we are finalizing plans to build a common database containing placebo arm data from industry trials and natural history data with an eye toward eliminating the placebo in future clinical trials.

Second, I have never had the opportunity to work with such a talented, effective and passionate bunch of people.

Ryan Fischer knows every single family in this community (it seems like) and can move effortlessly between IEP problems and congressional report language. Holly Peay is the brains behind the benefit-risk study, runs DuchenneConnect, and is the PI on a major grant to network DuchenneConnect through electronic health records with other similar registries—she brings incredible professionalism and rigor to everything she does. Kathi Kinnett is a nurse practitioner with many years of experience in the neuromuscular space and has managed, in a very short amount of time, to put in place a much-needed clinical center certification program to ensure that families know what care is offered at centers and how it aligns with the Duchenne Practice Parameters. Will Nolan is a gifted writer who handles communications and lots of other tasks so seamlessly that we frequently forget who makes it all happen. He may also well be the funniest person I know. Annie Kennedy, who was my colleague for ten years at MDA and my friend for even longer, joined the PPMD staff this year to help lead our advocacy efforts with Ryan Fischer. Anyone who knows Annie knows that we really scored when she signed on with PPMD—she would go to the ends of the earth for any of “her families” (that’s all of you, by the way) and she’s incredibly smart and driven to make a difference. Go Annie!

I also want to send a shout-out to our Grants Manager Elizabeth Habeeb-Louks who most of you don’t know, but who works hard behind the scenes to make sure that our scientific review process goes smoothly and that progress and expense reports are reviewed – and so much more. Liz and I worked together at MDA, too, so we have known one another a long time and I will miss her uncanny ability to read my mind. And then there’s Danielle Garrigan, who makes lots of things that happen at PPMD seem totally effortless, and Stephanie Matthes who is a creative genius with social media and anything web-related.

Of course Mama Kim (Kimberly Galberaith) orchestrates the conference every year and holds us all altogether with her ability to laugh at herself (and us) while gently keeping us all moving forward toward common goals. She frequently claims that she should really be “teaching little kids to dance”, but we all know that choreographing big kids is what she does best.

And then there’s Pat. Pat is the one who inspired most of us to be here in the first place—with her passion and vision for a world without Duchenne. Pat leads by example and no one works harder than she does (I have gotten texts from Pat at 3am before – yes, Pat, I don’t know what time zone you were in but it was 3am here and I’m sure it was important). From the moment I first came to PPMD, I appreciated the fact that I never needed to wonder if the organization was truly devoted to its mission. She is working, not just to honor the memory of her sons, but for all of your sons as well. And only Pat could say “An FDA Guidance? We can do that!” and execute it within six months. I wish the autism world had a Pat Furlong.

Many of you also know that John Porter, a former tenured professor and program director at the National Institutes of Neurological Disorders and Stroke is moving to PPMD as its CEO. John is extremely well-respected in this community (and others) and also a good friend of mine. I think the world of John and his signing on with PPMD was another huge “score” for the Duchenne community. John is the kind of guy who I think lays awake at night worrying about how best to do his job and advance therapeutics for Duchenne—in short exactly the kind of person you want at the helm along with Pat. In fact, John’s coming to PPMD was one of the things that allowed me to feel comfortable that I could move to MDF without leaving PPMD in a lurch.

So, this was a very long-winded goodbye. I will miss you all but won’t be truly “gone” as I will be serving on the research committee of PPMD’s Board and hope to continue to interact with many of you through Facebook and other avenues. I am also committed to helping PPMD find a really spectacular person to head the research program as Duchenne therapeutics start to become a reality and combination therapeutics take center stage. Meanwhile I am looking ahead to re-engaging with the myotonic dystrophy community, which is just now seeing its first new drugs enter clinical testing (and starting on that roller-coaster ride that the Duchenne community has become all too familiar with). That organization is headed by Molly White, another Type A personality who is driven to make a difference. Just my kind of organization.

Best wishes,


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Comment by carrie on January 5, 2015 at 2:23pm
We will miss you Sharon. Thanks for all of your hard work. It has made a difference and we are grateful for your efforts.

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