Welcoem to posts!!
in the future - u will be able to do some more stuff here,,,!! like pat catgirl- i mean um yeah... for now u can only see others's posts :c
A number of viewers have asked what is happening in terms of gene therapy for LGMD 2B/R2 (aka dysferlinopathy). That's of interest to me as well, as I have this disease! Today Sarepta released a letter about their plans for a "proof of concept" clinical trial in dysferlinopathy which they plan to start later this year. They briefly mentioned this in a letter a few months ago, but without much detail. The plan is to give gene therapy to a small number of patients intravenously, so that the AAVs carrying the dysferlin gene will reach all the muscles in the person's body. One can think of this as being analogous to the LGMD2E trial which dosed patients a few years ago. The reason for a proof-of-concept study specifically in 2B is that the dysferlin gene is large, so it has to be delivered in two pieces ("dual vector"), which isn't true of other gene therapies being developed for LGMDs or DMD.
The company emphasizes that all the details for the 2B/R2 trial haven't been finalized yet. The announcement is posted here: www.sarepta.com/community-bulletin-lgmd-2br2-commu…
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I posted a video a few months ago about a clinical trial on the compound ribitol in one form of Limb Girdle Muscular Dystrophy (LGMD2i, aka LGMDR9 FKRP related). Yesterday, in conjunction this the Muscular Dystrophy Association conference, some data from the clinical trial was released that looks very promising. I plan to make a video going over the new results, but here's the video going over the rationale for testing this compound in this particular subtype of LGMD.
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Hi everyone! Now that this channel has over 500 subscribers (thank you all!!!) the community blog feature is activated.
There are a couple videos that are coming out soon. One goes over announcements from a couple drug developers on planned gene therapy trials. The other discusses clinical trial readiness in Limb Girdle Muscular Dystrophies, which was recently brought up in a discussion between a drug company working on LGMDs and the biotech investment community.
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Welcome, dear viewers!
I'm a scientist who has worked on a few different topics in my career--currently for a nonprofit foundation working on treatments/cures for a particular type of muscular dystrophy (Limb Girdle Muscular Dystrophy 2B) which I myself have. Other topics I've explored in this channel include psychology/personality types/personal growth.