MLD Foundation

US Announces BRAIN initiative with $100m of 2014 funding

Leave a Comment / Policy, Research / MLD Foundation / April 2, 2013 / basic science, BRAIN, Brains for Brain, Collins, NIH, NINDSHuman Brain Project, Obama, research

President Obama today announced the Brain Research through Advancing Innovative Neurotechnologies (BRAIN) initiative and an anticipated $100M of US government funding in the next fiscal year. That is part of several hundred million more committed by private partners and foundations to this project to better understand how the brain works. http://www.nih.gov/science/brain/ NIH Director Francis Collins […]

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I’m Thankful …

Leave a Comment / Uncategorized / MLD Foundation / November 22, 2012

Today in the United States we celebrate Thanksgiving. Canada celebrated a few weeks ago and according to Wikipedia at least 7 other countries celebrate a similar holiday at various other times of the year. This is the 149th anniversary of formal observance of Thanksgiving Day

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Changed our blog subscription widget

Leave a Comment / Uncategorized / MLD Foundation / November 12, 2012

You may be receiving two notifications about this blog post. We changed our blog subscription tool a few minutes ago. I’ve manually copied all of the current followers to the new tool but I cannot unsubscribe you from the original tool (one of its many defects). You are the only one who can unsubscribe yourself

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Fiscal Cliff & Sequestration

Leave a Comment / Action Alert, Policy / MLD Foundation / November 11, 2012 / budget, budget control act, debt, deficit, fiscal cliff, NIH, sequester, sequestration, spending, taxes

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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RARE Patient Advocacy Summit – 2012

Leave a Comment / Advocacy, Awareness, Education, General, Newborn Screening, Policy, Research / MLD Foundation / November 11, 2012 / nbs, newborn screening, policy, RARE, research, summit, videos

I was pleased to be the organizer and host for the RARE Project | Global Genes RARE Patient Advocacy Summit on September 29th, 2012. The day-long event with 140 in attendance and over 120 viewing via a live webcast. Videos of the event are available below for viewing.

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PDUFA – 20 Disease Meeting followup … Firestorm – not!

Leave a Comment / Policy / MLD Foundation / November 6, 2012 / development, drug, FDA, firestorm, focused, NORD, patient, research

Thank you all for your response to my recent request to send letters to the FDA on the Patient-Focused Drug Development initiative last week. Quite a number of you (dozens) responded on behalf of rare disease in general and many on behalf of MLD or lysosomal disease specifically. As I mentioned in my prior post,

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Thanks for your support – be notified when the blog updates

Leave a Comment / Uncategorized / MLD Foundation / November 2, 2012

You all are awesome … we think there were several dozen letters sent to the FDA – just for MLD, and we know that the rare and lysosomal disease communities also contributed. Many of you have asked how you can get involved with awareness that has an impact … Every letter and phone call you

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FDA … 20 Diseases Being Chosen for PDUFA-V Meetings

4 Comments / Action Alert, Policy / MLD Foundation / October 30, 2012 / action alert, disease meetings, FDA, FDASIA, lysosomal, lysosomal disease, PDUFA, testimony

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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List of Rare Diseases

4 Comments / Awareness, Education, General, Research / MLD Foundation / October 30, 2012 / disease, leukodystrophy, list, lysosomal, NIH, ORDR, RARE, rare diseases

This list was prepared using data supplied by the NIH’s Office of Rare Disease Research in late 2011. Please note that your medical providers and insurance payors probably have their own lists of diseases they think are rare – this list is for quick reference only. Quick Jump: A B C D E F G

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Our new blog …

Leave a Comment / General / MLD Foundation / October 30, 2012 / discussion, FDA, nbs, newborn screening, NIH, policy, rare disease, research, welcome

Welcome to the MLD Foundation’s new blog! We’re excited to share about topics of interest to the MLD, lysosomal disease, leukodystrophy, and frankly, the entire rare disease community. Many of you in the general public and the MLD Family know us from the family support we provide to families affected by MLD … MLD Family

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