Posts tagged with fda

Did it Go Far Enough? FDA Issues Draft Guidance on Rare Disease Drug Development

Posted Saturday August 15, 2015 by Melissa

Yesterday, the FDA issued draft guidance on common issues related to rare...

Yesterday, the FDA issued draft guidance on common issues related to rare disease drug development. It is another importance event in the shift of thinking about how drugs are developed...

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FDA-Guidance

Immune Tolerance to Enzyme Replacement Therapies, Part 3: FDA/NORD Workshop on the Role of Immune Tolerance Induction

Posted Wednesday August 27, 2014 by Melissa

Immune response is one of the most important issue in enzyme replacement...

Immune response is one of the most important issue in enzyme replacement therapy today. At least it is to many patients. That’s why I’ve waited several months to publish this...

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Immune Tolerance to Enzyme Replacement Therapies, Part 2: Idursulfase

Posted Tuesday June 17, 2014 by Melissa

In Part 1 of this series, I explored some of the background...

In Part 1 of this series, I explored some of the background concepts related to immunogenicity. Now let’s begin examining immunogenicity in the context of a specific example – idursulfase...

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Elaprase

Immune Tolerance to Enzyme Replacement Therapies, Part 1: Background on Immunogenicity

Posted Friday June 13, 2014 by Melissa

It would not surprise me if many of you reading this have...

It would not surprise me if many of you reading this have never heard of immune tolerance induction. That may be, in large part, because prior to the last two...

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Antibodies photo under creative commons license from AJ Cann

#SaveJack, #BeBrave

Posted Friday January 17, 2014 by Melissa

I’ll never feel okay about kids dying. Never. And I’ll surely never...

I’ll never feel okay about kids dying. Never. And I’ll surely never feel okay about my best friend’s son dying. But in the whole convoluted process that is drug discovery,...

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What proof does the FDA want in a degenerative disease?

Posted Thursday January 16, 2014 by Melissa

Here is an example in Duchenne MD of why I spent last...

Here is an example in Duchenne MD of why I spent last week at the FDA. In a degenerative disease, if a drug can maintain abilities, why do children have...

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Sarepta Slide16

What is Compelling Evidence? The mother frogs speak

Posted Friday August 23, 2013 by Melissa

What have you been doing for the past 3 1/2 years? Having...

What have you been doing for the past 3 1/2 years? Having a few children? Learning a new language? Starting a new job? I’ve been watching lives being saved. Children...

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A Tale of Two Frogs: a rare disease story

Posted Tuesday July 30, 2013 by Melissa

“It’s a miracle!” she said, as she watched her son hop, ever...

“It’s a miracle!” she said, as she watched her son hop, ever so gently, for the first time. Having never had legs before, even hopping was quite a new endeavor...

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TaleofTwoFrogs

Intrathecal Trial Update – New port (hopefully)

Posted Wednesday June 27, 2012 by Melissa

So we recently learned about the new port-a-cath that is contemplated to...

So we recently learned about the new port-a-cath that is contemplated to be used in the next phases of the intrathecal clinical trial. If you’ve followed along, you’ll know that...

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Reversal of Fortune? Where Profits and Patients Collide

Posted Monday April 09, 2012 by Melissa

The rare disease world, and especially the non-precisely-defined ultra rare disease world,...

The rare disease world, and especially the non-precisely-defined ultra rare disease world, is a small one. You know the players. You meet over and again. Your pharma company knows your...

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Collision of two chess figures, pawn and king