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    • #23204
      Amanda
      Keymaster

      Good morning,

      Did anyone participate in the Toferson clinical trial? If so, what has your experience been like so far? Do you believe it has helped or hindered your condition? Did you continue the treatment after the trial was completed?

    • #23212
      Amanda
      Keymaster

      Updated:

      I was recently diagnosed, this past Friday, and this treatment was discussed because it targets the specific mutation that runs in my family. We’ve lost 15 family members to ALS, the last being this past February. I also have the mutation.

      I’ve been volunteering for medical research since 2010; however when the clinical trials started for Toferson, I did not meet the criteria for the trial. I was asking so that I could gain insight and information to help me make decisions about my future health care.

      Toferson was supposed to be reviewed by the FDA in January; however, Biogen submitted additional information to the FDA. The company put out a press release saying the the review date has been extended until the end of April.

      I will have to apply for extended access in order to be considered for the treatment. I was hoping that some of our members could provide me with information. I have learned since positing that many times when people participate in clinical trials they are legally obligated not to share or discuss information until the drug has gone through the FDA process.

      Amanda

      • This reply was modified 1 month, 3 weeks ago by Amanda.
      • #23274
        John Ceriotti
        Participant

        Amanda

        I started as one of the last participants in the “closed” portion of the study and then was allowed on the extended access or open access portion of the study.  I just received my 20th dose on Dec. 13th.  Myself and my wife (an RN) are very happy with the results of the Tofersen drug.  20 doses ago (one every 4 weeks via Lumbar Puncture) I was unable to lift my right arm above my head and continued to lose strength in my lower extremities.  I am very pleased with the results to date and actually started physical therapy at the Washington University Orthwein Center Sept 26th.  My results have been good to very good with regaining strength and the ability to perform many more physical things without having to tremendously focus as before the doses.  I do things physically without thought just as I did prior to my diagnosis.  I am still not walking but continue to gain more and more strength across all body parts.  In fact, my breathing capacity test increased to 48% from 38% over a 3 month period and tested on Nov. 13th or so.  my 27 year old son who lives out of town is just in awe and what I can do …like transfer from my chair to a PT mat, or from my chair to a toilet/shower chair or even at night when I transfer myself from my chair to my recliner without any assistance from others.  I feel stronger than I did 2 years ago.  Really I do! I am very pleased with the results and will continue to go to PT 2X per week looking for some more tangible results in the next 3 months…targeting the 6 month mark or so to be able to walk on my own power using a walker to start!  Get the drug , I believe it is worth the monthly LP until they come up with a preferred delivery method.  Email me with any other thoughts or questions…[email protected]

    • #23222
      Les Wood
      Participant

      Hi my name is Les Wood I was diagnosed in August 2012.i am on the Toferson treatment I am just starting my seventh year on it.I am the first person in the UK to be on the trial,it is now classed as a treatment,I feel the drug has helped me a great deal,I have endured around 75 lumber punctures during this time as of course that is how the drug is administered.there are I believe ten of us on the treatment which is at Sheffield Hallamshire Hospital.the staff there are brilliant and very professional.If any one wants any imformation on tis subject I am only to happy to help all I can

       

      • #23227
        Amanda
        Keymaster

        Les,
        Thank you so much for the information. Has it been approved as a treatment there? Tofersen was supposed to be reviewed in January byt the FDA, here in the United States. Biogen provided additional information so now it is expected to be reviewed in late April. my doctor’s have applied for early access to the treatment on my behalf. They are hopeful that will be approved and I could expect to start the treatments in Jan/February.

        How do you feel the following couple of days after the treatments? Are you getting them every 4 weeks or what is the schedule.

        I’m so happy for you and that you feel the treatments are helping you. I had no idea it had even been available anywhere for 7 years!! Any breakthroughs will help the medical professionals figure out how to help all pALS!

        Amanda

        • #23230
          Tina Johnson
          Participant

          Hi Amanda,

          I’m also interested in the tofersen treatment. Can I ask you what state you live in? Also where are you being treated at?

          Tina

        • #23275
          John Ceriotti
          Participant

          Tina

          Just to assist although the question was to Les…I am in StLouis and being treated at Washington University Medical Center where the creator of the drug Dr. Timothy Miller is the Chair of the department with his PI Dr Robert Bucelli who is Vice Chair!!

        • #23283
          Tina Johnson
          Participant

          Thank You, John

          Sounds awesome what it has done for you.

          I am 40 and walking with a cane, I can’t lift up my left arm very high anymore and now it’s starting in my right. I am also doing PT and exercising at home as much as possible.

          I talked to my Neurologist and they put me on top of the list for Tofersen. I am just praying I am able to get it.

          Thank you for posting!

          Tina Johnson

           

        • #23286
          John Ceriotti
          Participant

          Tina

          Any supplements as I do take (7) to help with body health!

          My wife who is an RN-BSN did a bunch of research 2 1/2 years ago when I was diagnosed and found a good amount of info concerning what supplements showed any type of positive or non-harmful results.

        • #23290
          Tina Johnson
          Participant

          Hi John,

          Would you be able to let me know what the supplements you take? Either on here or email?

          My email is [email protected]

          Thank you!

    • #23229
      Suzanne Gallo
      Participant

      Amanda

      i am so very very sorry that you have been diagnosed with ALS. Please know that I am extremely sad. I have been following your column sincd my husband was diagnosed about 3 years ago.
      my father passed from ALS 6 years ago. Not familiar, but super lousy, just the same.
      I suggest you contact Ron Hoffman, at CCALS.org. Please do this, to broaden awareness of patient, family, and caregiver care.
      you must do as you wish, clinically, but as far as genuine care for you- and, your caregivers, please reach out to CCALS.
      They have been the world to my PALS and I.
      suzanne

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