Takeda announces preliminary ERT trial results Early results not as positive as hoped for Takeda Pharmaceuticals announced that a preliminary look at the results of their recently completed phase-IIb trial of their TAK-611 ERT (enzyme replacement therapy) for MLD “topline results … did not meet primary and secondary endpoints.” The company is analyzing the results and […]
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 »
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
80% of the 7,000 Rare Diseases are genetic in nature. While most rare diseases show symptoms early in life, many are later onset diseases because our genetics are always with us. Genetic inheritance patterns can vary from single gene autosomal recessive (like MLD) where 50% of offspring are carriers, 25% are affected, and 25% are free of the
A very interesting question came up today on the MLD Family Discussion List™ (a private list we run for MLD primary caregivers). I thought it might inspire some thinking so I am sharing my answer here as well: I have a question and I don’t know if anyone has an answer but I am wondering if anyone
Are normal enzyme levels enough to stabilize MLD? Read More »
