Tag Archive: rare diseases

Jun
24

BIO2016 and getting Big Pharma to listen to the patient’s voice..

Its hard to believe that a couple of weeks ago BIO2016 was coming to an end. After 5 days in San Francisco my legs were tired, my voice was rough, and I was tired of boxed lunches and eating dinner at crazy irregular times – generally very late. Its a hard life! I was at …

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Dec
28

2015 in review

2015 saw the continuation of some research¬†themes from 2014, namely some work on Ebola, rare diseases and Bayesian models for neglected diseases. At the end of the year several papers are in press for next year so more to come. As usual there were some ups and downs in getting some of these to see …

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Sep
11

C&EN’s Advances in Drug Discovery and Development

Next Thursday at 10am EST I will be giving a virtual seminar at this virtual symposium – you can find out more and see all the other speakers too. This is a first for me – So I hope for some interaction via Twitter #CENSYM15 Slides are done, now I just have to prepare what …

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Jul
29

Wearing many hats

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 …

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Jul
24

Targeted Drug Development : The FDA perspective – A more approachable regulator?

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 …

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Jun
01

Find out more about Sanfilippo Syndrome and Charcot-Marie-Tooth

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 …

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May
07

The Necessary – a provocative vision of the scientific future

I was asked by Barry Bunin a few months ago to provide a short presentation on a vision for the future of drug discovery which I finally gave this week. After thinking about this for a while and chatting with a few friends including Alex Clark, – I wondered what was necessary in the future …

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Dec
15

A year in publications – 2014

A year in collaborative publications, the ups and downs and a few random comments as well (with a big thanks to all involved): 1. Ekins S and Freundlich JS and Coffee M, A common feature pharmacophore for FDA-approved drugs inhibiting the Ebola virus, F1000research, 3: 277, 2014. This came initially from a Twitter exchange of …

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Jun
19

Itching to get back in the lab

I thought I might never want to do this again, but a confluence of circumstances is making me want to do something. I am itching to get back in the lab. One day. My last wet lab experiment was in 1998, sure I have managed others since then that did ¬†experiments in my lab, or …

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Jun
11

Rare Occurrence Reception

Jill Wood from Jonah’s Just Begun has a cocktail event planned in New York next week to raise money to fund a postdoc in one of the labs doing Sanfilippo syndrome research. This is obviously something I am very close too.¬† It just shows the willingness and generosity of those involved to help out (Jonny …

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