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Research

FDA BLA Filing Completed for MLD Gene Therapy

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FDA Filing Completed for MLD Gene Therapy Orchard Therapeutics requesting Priority Review of BLA Orchard Therapeutics has announced the completion of their BLA filing and a request for Priority Review with the US FDA for OTL-200, the gene therapy approved as Libmeldy in Europe.  Their “rolling-BLA” filings started last quarter. The BLA is a “Biologics License Application“, the process used to get an […]

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Takeda Pharmaceuticals logo - 2023

Takeda announces preliminary ERT trial results

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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

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FDA monument

Orchard Starts Filings with FDA for MLD Gene Therapy

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Orchard Starts FDA MLD Gene Therapy Filings Orchard Therapeutics has announced they have filed the first module of their rolling BLA  with the US FDA  for OTL-200, the gene therapy approved as Libmeldy in Europe.  The BLA is a “Biologics License Application” that will be reviewed by the FDA. A “rolling BLA” allows Orchard to file the application in multiple parts, which

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#GivingTuesday

Your Giving Keeps Us Going – and directly helps families! #GivingTuesday

Leave a Comment / Advocacy, General, Newborn Screening, Research / /

#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

Your Giving Keeps Us Going – and directly helps families! #GivingTuesday Read More »

#GivingTuesday

Your Giving Keeps Us Going and directly helps families! #GivingTuesday

Leave a Comment / Advocacy, General, Newborn Screening, Research / /

#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

Your Giving Keeps Us Going and directly helps families! #GivingTuesday Read More »

#GivingTuesday

Your Giving Keeps Us Going! #GivingTuesday

Leave a Comment / Advocacy, General, Newborn Screening, Research / /

#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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Tissue Donations … The Ultimate Gift!

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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

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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

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

MLD Newborn Screening Pilot Study – Announcement

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Today is Rare Disease Day. It’s an appropriate day to announce we have come one step closer to a MLD Newborn Screen! The pilot study to test the newly developed MLD Newborn assay/technique on current newborn babies born in Washington state will be starting within the next three months once testing lab equipment is in place.

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