Ibudalist, PrimeC, NP001, Amboxol

[JAM]

I’m sure there are other medications in trial/experimental, however we need to move forward and try SOMETHING. If any PALS, CALS is currently using, or has tried any of the above drugs please respond with history, experience, how you got the medication etc. thank you in advance.

 

[Brad H]

I began in the COMBAT trial (Ibudilast) in late 2022. This was through my clinic at Univ of Virginia. I graduated out of the trial and opted for the open label. Once open label expired, the sponsor offered and with the help from my care team at the clinic, they applied and I was granted FDA Compassionate Use for Ibudilast. I am still taking twice daily. No side effects but I have no idea if it’s working. I’m also taking Riluzole- and had been on Relyvrio too. Still progressing- coming up on 3 years since symptoms.

 

[JAM]

Thank you Brad. I appreciate the information. What was you initial onset? Where are you in this journey. Are you still able to walk? Breathe without assistance?

 

[Brad H]

While it’s hard to say where I am in this journey, I would say my personal feeling is that I’m progressing about average. Limb onset in one hand/wrist. Within 10 months noticing foot drop, it has now progressed to include pulmonary function, neck droop, other arm, both shoulders are “pulling” unless I keep arms on pillows. I have lots of braces that help support all the floppy parts. I’m in power chair or bed at all times, no walking, stairs or driving. Dressing and hygiene with assistance. Am blessed that I can still chew and swallow. Speech still works although with lungs affected, very scratchy and whispery. I do NIV all night and as needed some days. All in all, I believe I can still do more things than I can’t.

 

[JAM]

Thank you for sharing. I appreciate it very much.