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GSK Launches Phase 3 Cryopreserved Gene Therapy Clinical Trial

2 Comments / General / MLD Foundation / December 14, 2017

New Clinical Trial for MLD GSK has just launched a Phase 3 clinical trial to study the efficacy of a cryopreserved formulation of their gene therapy, i.e. freezing the modified cells before transfusion. Recruiting of pre-symptomatic late infantile and early juvenile patients will start immediately. This study still requires patients to travel to Milano, Italy […]

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Your Giving Keeps Us Going! #GivingTuesday

Leave a Comment / Advocacy, General, Newborn Screening, Research / MLD Foundation / November 27, 2017

#GivingTuesday is Here! Here in the US we celebrated Thanksgiving this past weekend. It’s a time of family, thanks, and giving. Most of us “gave” at the mall on Black Friday or online on Cyber Monday … but now it’s #GivingTuesday, an annual day where the world comes together to give. November is MLD Foundation’s

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rare disease week Capitol Hill group photo 2017

Our first Rare Disease Week in DC

1 Comment / Advocacy, Awareness, Education, General, Policy / Tony Hodgson / March 21, 2017 / DC, Hodgson, MA, NC, Pierce, Rare Disease Week, RDLA, RDW, Washington DC

At the 2016 MLD Family Conference a conversation with Dean led to a decision to attend the Rare Disease week. It was a first visit for wife Nancy and I to Rare Disease week and a pleasure for us to assist daughter Michelle in attending this year. Last year she had planned to attend but

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A Call for Rare Facts Unity & Consistency

2 Comments / Advocacy, Awareness, Education, General / MLD Foundation / March 4, 2017 / 1 in 10, 7000, advocacy, awareness, facts, statistics

It’s Our Opinion … I presented this poster at the NIH’s Rare Disease Day event earlier this week (2/27/17). In short, to maximize impact and credibility, we are asking all organizations – government, advocacy, industry/pharma, academia, etc. to be consistent with their public sharing of Rare Disease facts. I realize that sometimes we must be very scientific about

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Tissue Donations … The Ultimate Gift!

Leave a Comment / Awareness, Compassion, General, Research / MLD Foundation / January 9, 2017 / bank, BRAIN, donation, donor, skin, tissue

A MLD mom recently asked about donating her son’s brain and other tissue to research after he passed. There is no more personal or profound donation that can be made to advance MLD research. This post is a lightly edited copy of my response to this special MLD mom … It’s always difficult to talk about tissue

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Capitol Hill, BIO, 21st Century Cures, Airbnb, and one more thing … Reflecting on my DC week

Leave a Comment / Advocacy, Awareness, Compassion, General, Policy, Research / MLD Foundation / November 6, 2016 / BIO, Congress, CURES, curesNOW, Eden, FMLD, MLD, policy, RarePolicy

The first rule of blogging is to post good content on a regular basis. The second is to keep your posts short, to the point and to not mix multiple topics into one post. Oh boy, am I in trouble … My 6th trip to DC this year ended reflecting at 35,000′ with a Crater Lake Hazelnut Espresso Vodka courtesy of

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Rare Disease Week in DC … a parent’s perspective

2 Comments / Advocacy, Compassion, Education, General / MLD Foundation / March 14, 2016 / Brown, Capitol Hill, rare disease, RDD, RDW, Washington DC

For those of you who know me, I have been a teaching computer skills to middle school kids for 17 years!. While I love school now as a teacher, I did not enjoy all aspects of school as a student. My favorite classes were Science and Gym! Social Studies, not so much! Shhhhh….. I really

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MLD Foundation – Very Active This Rare Disease Day (2/29)

1 Comment / Advocacy, Awareness, Education, General, Newborn Screening / MLD Foundation / February 29, 2016 / Capitol Hill, Caucus, legislative conference, NIH, rare disease day, RUSP roundtable

February 29th is Rare Disease Day this year. 1 in 10 – 30 million Americans – have one of the 7,000 rare diseases. On this rare day take a look around – who is it in your circle that has a rare disease? MLD Foundation is very busy this Rare Disease Day not only on

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Hear hoofbeats? Think Zebras … it could be a Rare Disease!

4 Comments / Advocacy, Awareness, Compassion, Education, General / MLD Foundation / February 7, 2016

When we hear “clippity clop” our brains immediately think horses. Zebra is the American medical slang for arriving at an exotic medical diagnosis when a more commonplace explanation is more likely.[1] It is shorthand for the aphorism coined in the late 1940s by Dr. Theodore Woodward, professor at the University of Maryland School of Medicine, who instructed his medical interns: “When you hear hoofbeats, think of horses not zebras“.[2] Since horses are common

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50% of those with Rare Disease are Children, 30% will not live to age 5

1 Comment / Awareness, Education, General / MLD Foundation / February 6, 2016 / children, death, rare disease

50% of those affected with Rare Disease are Children! Of those children, 30% will not live to see their 5th birthday. Rare Disease is a leading killer of our children. Nearly a third of the children with rare disease will not live to see their 5th birthday. Rare Disease is responsible for 35% of all deaths (of

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