1 person in every 10 people has a Rare Disease. 30 million Americans … 30 million Europeans … 350 million around the globe … all with Rare Disease. Pause for a moment and think about your family and those that live around you … many cancers and most childhood diseases are Rare Diseases. On […]
1 in 10 Have a Rare Disease Read More »
My name is Tarryn and my older sister Wanita was diagnosed with MLD in 2007. She was 16 and I was 14 when she passed away and passed in 2010 from juvenile MLD. I live in South Africa. She was so loving and so caring. Wanita was a very friendly person who loved everybody she
Wanita … How a younger sister sees MLD Read More »
Happy New Year! As we head out of the holiday season and into a new year, it is a time that we reflect on life, cherish family, and count our blessings for all that we hold close to our hearts. Holiday music has been playing everywhere you go for the past month; while I enjoy
New Year’s Reflections … and Highlights from December Meeting in Boston Read More »
As you read this most of the MLD Family™ is up and in the midst of their Christmas day – we have a Hawaiian family that is (hopefully) still snoozing! It’s Christmas all over the world … from New Zealand & Australia through SE and central Asia, Europe, Africa, North, Central and South America the
Registration for our 2105 MLD Family Conference in Newark Delaware is underway and closes in just a couple of weeks. If you are a MLD Family we’re anxious to have you register. Many of your MLD Family will be there to meet, share, and socialize with. Please bring your MLD loved one with you – we not only want
Mld Family Conference™ – Register Now! Read More »
Earlier last month we had the opportunity to see several presentations about the Leukodystrophy Center of Excellence (CoE) at Children’s Hospital of Philadelphia (CHOP), which opened today, May 1st. The mission of the [CHOP Leukodystrophy CoE] center is “to deliver cutting-edge, integrated, multidisciplinary clinical care, diagnostic evaluation, and therapeutics to infants, children and youth with inherited white matter disease.”
Centers of Excellence for Leukodystrophies and Lysosomal Disease Read More »
Today, February 28th, is Rare Disease Day (actually RDD is the 29th – but we don’t have one of those this year!). Today we celebrate and recognize the 1 in 10 of us that have one of the 7,000+ rare diseases … over 30 million here in the US and 350M worldwide, what would be
Reflections on Rare Disease Week 2015 Read More »
A meeting of researchers, clinicians, industry and academia was convened by the MLD Foundation on June 24th in Washington, DC to discuss the openNHS Manifesto we wrote about in this blog post. Since a NHS is not a therapy, NHS participants have historically be giving time, energy, and effort, not to mention exposing their MLD loved ones to occasional invasive
openNHS Manifesto – Meeting Report Read More »
The Enzyme Replacement Therapy HGT-1110 Phase I/II clinical trial has been fully recruited. Patients are participating from three continents; Europe, South America, and Australia. The primary purpose of a Phase I/II clinical trial is to study safety and to establish a target dosage for further study. The trial lasts 40-weeks so the last patient should complete their
ERT Phase I/II Clinical Trial Recruiting Completed Read More »
Great Patient Focused Drug Development (PFDD) meeting at the FDA Tuesday discussing patients perspective on the neurological inborn errors of metabolism (IEM). Dean Suhr, president of the MLD Foundation, spoke on the second panel of the day and was able to both share and stir the pot a bit with regard to some of the patient perspectives on
Report from FDA PFFD IEM meeting Read More »
