Sep
09
Previously I had written about the need to find patients for Sanfilippo Syndrome IIIC and IIID to join a registry and take part in a Natural History Study. In fact I keep writing about it in as many places as I can. In addition we are actively working with academics to develop treatments so it …
Aug
20
I am just back from a very busy trip to Boston for the ACS. It was great to meet up with many people I see only rarely, at these meetings as well as meet new people including one with a molecule tattoo that got a lot of interest on Twitter . I gave 5 talks …
Aug
14
A busy day before going to the ACS. We have put a couple of similar posts (here and here) out in an attempt to try to identify some Sanfilippo type D (MPS IIID) patients or at the very least to get their families to register in the registry . This is like finding a needle …
Aug
11
Well next week it is the ACS in Boston and of course I leave it to the week before to pull everything together. I initially put up here my listing of abstracts titles etc back in May. This has now been updated a bit to include additional talk and poster showings as well as times …
Jul
29
I wear a few different hats and yesterday I was CEO, working on the commercialization plan for a phase II grant for a rare disease. To this point the big selling point was getting a Rare Pediatric Disease Priority Review Voucher but this is a challenge because its unclear if there will be more of …
Jul
24
As I am slowly catching up on my reading and I came across this new white paper type doc from the FDA which came out a few weeks ago called Targeted Drug Development: Why are Many Diseases Lagging Behind? It makes for an interesting read and of course conjures up images of diseases on a …
Jul
09
Its been a pretty hectic year to date with several grants coming to their conclusion and the need for writing up final reports and manuscripts. In addition it has provided just a little time for reflection. Pretty much everything I am involved in is a collaboration of some sort so I am just a tiny …
Jun
01
A couple of diseases I work on are described in two of recent newsletters. First up is Sanfilippo Syndrome, which is described in the Lysosomal Disease Network’s newsletter Indications. This describes Jonah’s Just Begun and Phoenix Nest and was written By Jill Wood. I did a bit of proof reading of this article so I …
May
26
Ever since embarking on my pretty unusual career path in leaving big pharma in 2001, I have been faced with several forks in the road, hard decisions on which way to go. To join a software company or not? work with university spin out? – some were good decisions others less so. Since 2008 I …
May
12
It is that time of the year when we get those automated emails telling us our abstracts have been scheduled for the next ACS in August. In addition to these I will be participating in a careers panel for undergraduates which will be interesting – hopefully to inspire the next generation that there is lots …
