Today is the start of the 17th annual WorldSymposium™ Lysosomal Disease annual meeting. WORLD is the preeminent lysosomal disease research and translational science meeting. Over the last decade and a half, its attendance has grown from a couple of hundred to well over 2,000. MLD Foundation has attended 16 of the 17 WORLD meetings. Unfortunately, […]
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
We heard earlier this week that nearly 4 in 5 people are already looking forward to putting 2020 behind us. The pandemic (family safety, work, school, loss of loved ones, individual and community response, social isolation, etc.), US elections, income, health, … it is affected every one of us, and it’s overwhelming. But we are
Gene therapy for early-onset MLD patients passed a huge milestone yesterday when the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending full, or standard, marketing authorization for Libmeldy, by the European Commission (EC), which has the authority to grant marketing authorization for Libmeldy* in the European
Gene Therapy Recommended for Full EU EC Marketing Authorization Review Read More »
Warren Joseph shared a name with his father and grandfather. His first word was ball, and he ate his peas one at a time. For this, he earned the nickname Birdie. He was just like every other happy toddler, with aunts and uncles, brothers and sisters, parents and grandparents that adored him. The moment we
Hello family, friends, and those looking to connect and share stories. My name is Amy I am the mother of three adorable children John, Anna, and Andrew, all of whom are about 1 year apart in age. Sorry in advance I am a pragmatic writer. But do keep in mind this whole experience, even til
Orchard Therapeutics made some significant corporate changes … MLD gene therapy remains their lead program.
Update – Orchard Therapeutics Gene Therapy Program Read More »
Trent Alexander McKenzie was born a normal happy boy. He was a delight to everyone who knew him. He was funny, smart, and dearly loved by his family. We were all devastated when he began to lose abilities and was diagnosed with juvenile MLD in 2000 at the age of 10. He lived 19 years
