Action Alert

Giving Tuesday – It’s Our Day

Did you know that today, Tuesday, December 1st, is Giving Tuesday? After a month of “Black Friday” and yesterday’s “Cyber Monday” frenzy, it’s a day to focus on our loved ones! We aren’t asking for your donations … MLD Foundation knows that MLD families have their hands full with their loved ones so for the […]

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Say it’s not true … New Zealand proposes NO access to an “effective therapy” for a rare disease

Has my Sunday morning coffee not kicked in?  Am I reading this wrong? Tell me it’s not true … Alexion has an “effective therapy” for paroxysmal nocturnal haemoglobinuria New Zealand is proposing to decline access to therapy for its citizens  … not because it does not work – they acknowledge it is an “effective therapy” – rather, they

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Fiscal Cliff & Sequestration

Backgrounder … What is this & why do or should I care? Fiscal cliff – the effect of a series of enacted legislation which, if unchanged, will result in tax increases, spending cuts, and a corresponding reduction in the budget deficit. These laws include tax increases due to the expiration of the Tax Relief, Unemployment Insurance

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FDA … 20 Diseases Being Chosen for PDUFA-V Meetings

Last Thursday, October 25th, I attended the FDA’s Patient-Focused Drug Development public meeting in Washington, DC. This meeting was required by the recently passed FDASIA/PDUFA-V legislation (see Abbreviation Decoder at end of post) and requires the FDA to hold 20 disease-specific meetings over the next 5 years to discuss topics such as: “the impact of the disease on patients,

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