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    • #24969
      Kevin Schaefer

      Happy Tuesday!

      Check out Michael’s latest video here. He talks about Scholar Rock’s experimental therapy SRK-015 and its potential impact on the SMA community. This episode is based on an article from our main website, which you can read here. Hope you enjoy all watching this one!

      Do you have any questions regarding this therapy?

    • #24989
      Lupa F

      Why is the video titled “Unbelievable Results So Far…” when there’s been no data released from the trial yet?

    • #24990
      Kevin Schaefer

      Hey Lupa, thanks for commenting!

      While we don’t have a ton of data regarding this treatment yet, it’s promising that the FDA is showing a lot of interest in it. Plus, it works at the muscular level, and could positively impact the strength of trial participants. Hence, we wanted to emphasize that what we do know is really exciting.

    • #24992
      Lupa F

      I wouldn’t read much into the FDA adding it to the Rare Pediatric Disease Designation. It’s an incentive program to encourage companies to develop drugs for pediatric rare diseases, it doesn’t give any indication about how the FDA feels about a particular drug. A company just has to apply for it and if the drug meets the criteria it gets it. The program gives the company an incentive to apply for this by shortening the review process for the drug in the application and also gives them another priority review voucher if the first drug gets approved.

      From the original press release: “If a biologics license application (BLA) for SRK-015 for the treatment of SMA is approved by the FDA, Scholar Rock may be eligible to receive a priority review voucher, which may be redeemed to obtain priority review for any subsequent marketing application or be sold or transferred.”

      I don’t have a problem with the article Michael talks about, but the video title is extremely misleading as there are no meaningful trial results published yet.


      • This reply was modified 8 months, 1 week ago by Lupa F.
    • #24995
      Michael Morale

      Lupa –

      You actually pose a very good question with regards to the data. Even though no formalized data has been released, Scholar Rock has mentioned on quite a few occasions about how muscle targeted therapies will help those of us with SMA.

      All of us know that Spinraza and Evrysdi both work at the cellular level, and while there was a lot of hope and anticipation that this would allow us to get stronger, both of these treatments do not address previous damage that has already taking place in our bodies. These treatments will either slow the progression or possibly stop the progression from getting any worse, but until a muscle directed targeted therapy is passed by the FDA, this is the only thing that we have to work with.

      Being that SRK-015 is the first muscle targeted therapy, all of us are hoping that it will allow us to actually build muscle mass. If this actually comes to fruition, I’ve heard from numerous people that this could be our greatest chance at a combination therapy. If we could take Spinraza or Evrysdi, which work at the cellular level, and also take SRK-015, which works at the muscular level, this will give all of us who suffer from SMA the greatest chance at possibly building muscle mass, and we may even begin to see some repair from previous damage that had already taken place in our bodies.

      I hope this explains it a little bit better, and feel free to let us know if you have any other questions. Thank you.

    • #24996
      Lupa F

      I understand what the drug is aiming to do. That’s not my issue. You title your video “Unbelievable results so far…”. What results are you talking about? There are no results in the article you’re reading from.

    • #24997
      Lupa F

      There have been 2 other myostatin inhibitor drug trials (for Duchene MD) that were both discontinued once trial data was available because they ended up not being effective.

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