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Friday, April 20, 2012

Insight from a good friend. Justine gets it, she really gets it.

A few months ago, I stepped off an elevator in Brooklyn to find a beautiful blond haired boy with an infectious smile singing a song as I walked towards him. You would never know that this was the first time that we had met. You would never know that one year ago we were total strangers. You would never know that a rare genetic disease could end his life, long before his time. But, then you would surely not know Jill, his mother, who is fighting with every ounce of fight that a mother has to make sure that this does not happen.

Friday, April 13, 2012

Insult to Injury #PDUFA #FAST

You all know how hard our rare disease community has been working on getting this language added to PDUFA:

"Considerations. – In developing the guidance . . . . the Secretary shall consider . . . . for drugs designated for a rare disease or condition under section 526 of the Federal, Food, Drug, and Cosmetic Act; and (2)how to incorporate novel approaches to the review of surrogate endpoints based on pathophysiologic and pharmacologic evidence in such guidance, especially in instances where the low prevalence of a disease renders the existence or collection of other types of data unlikely or impractical"

Wednesday, April 4, 2012

This week/month/year...no rest for the wicked.



This week/month/year... no rest for the wicked.

Monday we kicked off the “Pay it Forward” campaign. The HS students at LIFE academy for Music and Film in Brooklyn has honored JJB, by asking us to be the recipients of their Pay it Forward campaign. The students at LIFE academy came up with a great idea to bring their community closer while helping others. The students are planning several fundraising events. They’re selling colorful paper hands (helping hands for Jonah), then placing the signed hands down their hallway at school. On the other side of the hallway the kids are placing paper bricks, creating a brick wall. On each brick the student writes a good deed that they performed. The choir is putting on a a talent show modeled after “America has Talent.” Their version “LIFE academy has talent” (view the event here) Many of the students will be sending out Cause wishes in honor of JJB-Helping Hands for Jonah. The HS students will present JJB with a check on graduation day. Their goal is drum roll please... $10,000! JJB set up a cause wish as well (view it here), to help them out with their ambitious goal.