Category Archive: Rare disease

Sep
20

Progress Through Partnership: The NINDS 2016 Nonprofit Forum

Last tuesday and wednesday (Sept 13-14th)  I was very honored to be an attendee at the “Progress Through Partnership: The NINDS 2016 Nonprofit Forum“. My role was to provide an industry perspective on several panels (alongside Dr. Ronald Marcus, Cerecor). There were a large number of rare disease (patients or parent) advocates, academic scientists and …

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Aug
04

Gearing up the new company

Well, its been quiet for several weeks as I had a few projects to complete. One was the final report for an SBIR and in addition I have been curating a special edition of Drug Discovery Today focused on Tuberculosis as well as writing grants (I am always writing grants). So lots of writing basically …

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Jul
11

Experience with Reddit Science AMA

Today I took part in a Reddit Science Ask Me Anything (AMA). I had been invited to do this by the nice folks at Springer so I chose rare diseases as my topic and had 100s of questions to answer. This naturally took far longer than 1hr to answer. Generally a good mix of topics …

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May
31

New grants for Phoenix Nest Inc. and LABioMed for Sanfilippo Syndrome treatment development

I can now report some very good news for Phoenix Nest Inc  in our efforts with our wonderful collaborators at LABioMed – we now have a couple of grants to work on treatments for Sanfilippo B and D.  It would be great if we could find VC or angels that would help us get these …

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Mar
18

10 more years of the Pediatric rare disease priority review voucher

I just saw an email from Nancy Goodman and The Kids v Cancer Team describing the passing of legislation that will lead to the extension of the pediatric rare disease priority review voucher. This is a topic I have covered here and most recently in our recent publication with Jill Wood. In full disclosure I …

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Jan
26

Creating a rapid response – relevant to Zika virus

I have been following the news on the Zika virus over the last few weeks as it has spread with travelers returning from trips and women giving birth to children with microcephaly. Along with it is a spreading underlying fear of where it will lead. Its not so long ago that Ebola, was grabbing headlines. …

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Jan
15

ACS San Diego Talks

My schedule for the ACS in San Diego came through today – if you want to catchup please get in touch! PAPER ID: 2393678 PAPER TITLE: Virtual collaborations for developing Sanfilippo syndrome treatments on a shoestring (final paper number: SCHB 5) DIVISION: Division of Small Chemical Businesses SESSION: Start-up Businesses in Drug Discovery SESSION TIME: …

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Dec
15

Pitching a rare disease treatment, vouchers and more underscores need for collaboration

Over the last few weeks I have been pitching the Phoenix Nest enzyme replacement work with LaBioMed in New York, working on proofs for several papers (here and here) and putting a blog together on the need for the rare pediatric disease priority review voucher. This is in addition to the usual grant writing projects …

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Nov
12

Giving Tuesday- Giving changed me too

I wanted to help Jonah’s Just Begun so I contributed this to Giving Tuesday..please give it a vote.. November marks the anniversary four years ago that led to my commitment to try to help families find the cure for rare diseases that affect their children. One meeting at a conference led to a cascade of …

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Sep
09

Raising awareness for Sanfilippo Syndrome IIIC and IIID – could Microsoft help us find our patients?

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 …

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