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 still thankful…
… not for MLD, not for loss, but for all of you – our friends, family, the MLD Family™, the greater MLD community, the MLD researchers and clinicians, regulators, policymakers, and the rare disease ecosystem who supports us.
The MLD Family™ …
… is supporting each other. There is advice, compassion, and the sharing of excess or needed supplies. We can’t meet in person, but we’re connecting with regular men’s, women’s, and now couple’s calls and so much more as we adapt to new ways of connecting.
MLD Research is progressing …
Orchard Therapeutics has achieved EMA/CHMP recommendation for approval of gene therapy in Europe. They have also filed an IND with the US FDA, a key stepping stone formalizing communications with the FDA as they move one step closer to US review and approval.
Takeda continues its ERT clinical trial. Homology Medicine continues to make pre-clinical progress with their gene therapy. And we are working with several other biopharma companies, too!
MLD Newborn Screening …
We have to diagnose children earlier in order for them to have good viable life-saving therapy options!
The MLD Newborn Screening program is making great progress. New York is right on the cusp of launching an MLD pilot study into parts of their state. The MD NBS Expert Advisory Group is meeting monthly. Many of you are participating with the Working Focus Groups – you can still join us for our December meetings!
MLD Cinical Care Continues to Improve …
We are blessed to have many wonderful and knowledgable MLD clinical researchers … we still need more, but those who do support our community are open, supportive, and often go the extra mile as they care for our loved ones in person or remotely. We worry about their health as they are called into care situations where additional COVID resources are necessary – let’s all keep doing our part to keep these COVID care needs minimized and our clinicians safe!
In 2020 we started to lay the foundation for creating an MLD Standard of Care so we can all more easily get approval and reimbursement for better, more consistent, and more appropriate MLD clinical care.
Let’s keep connected …
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