I have friends saying that it's too much they know they can't afford it and they're just going to go to bed when their three four dap runs [Music] out so you have a situation where the product that you have to take for your health will now cost you an unaffordable $375,000 a year yes Rebecca as I standard you have been diagnosed with a rare neuromuscular disease known as LMS is that correct that is correct okay can you tell us a little bit about the disease and what's been going on recently that has gotten you so
concerned in 2005 I got really sick and my body turned against itself LMS can be an autoimmune disorder as well as being caused by cancer limbs is a pre-neural junction problem where it's not getting enough energy to give your muscles power so what happens is I can go go go for a while but if I sit down I just crash I was sleeping sometimes 18 of the 24 hours a day just to be able to be around my family you're a mom and that doesn't work when you have kids huh it does not it does
not at all so you came down with this illness which has really impacted your life but a couple of years ago you were put on a drug which has as I understand it worked well it was amazing within like 15 to 30 minutes I felt like this huge weight had been lifted off my body that's how how immediate it was I mean it truly is a miracle drug it's a miracle drug and it help you and we think many many hundreds of other Americans are suffering from how much did it cost you to get this
medicine what happened there's a small father and daughter Pharmacy called Jacobus Pharmaceuticals who were asked by the MDA muscular distrophy Association 20 years ago to make something that had been discovered in Europe they made it it was so cheap to make and there were so few of us with LMS that they decided to give it away for free on a compassionate use basis it cost me nothing for the last 2 years I have not paid a penny for it Dr Jacobus and all the others there have just seen it as a way to do the
right thing to help people out like myself who without it would be disabled so Jacobus was a pharmaceutical company that did exactly the right thing they reached out yes just a huge help to you and what's happened recently for the past couple years there have been an uneasiness that there's this other company Catalyst from ticals who bought for $5 million a similar type of molecular structure a drug like Jacobus was making of the 34 dap and they called it fur daps it was basically the same as 34 dap they pushed through their trials and the
long and short of it is they got FDA approval in November of 2018 and in their investor call on December 13th 2018 they said that the average cost for one LMS patient per year was going to be $375,000 dollars per person per year which I would imagine is something that is unaffordable to the vast majority of the people correct a so-called miracle drug once given to people for free may now cost hundreds of thousands of dollars all right and this company is catalyst the gentleman who is head of catalyst Catalyst Pharmaceuticals unlicensed drugs to treat
rare neuromuscular diseases what would you say to that gentleman I'd ask him what his intent when he bought the for formula from biomarin in Europe for fur apps was his intent to help us more in the lamber eatan Community or was his intent to make money because if that was his intention we were all doing just fine with our three4 da at Jacobus and had wonderful care there my understanding is that it costs so little to make this actual medication so again how are they justifying that and they bought it so they get orphan drug
status and that allows them seven years of exclusivity so jacoba has had to stop producing three4 da I suspect the anxiety and the stress are not helping your condition yes it's not at all actually stress is something that other than using your muscles too much can exacerbate the symptoms of Limbs so many people are even saying they're going to give up sorry if that breaks my heart cuz that shouldn't be you know it shouldn't be and we're going to do everything possible to make sure that it doesn't happen it really is unimaginable to know that
you're on a medicine which allows you to live a fairly normal life yes yes it does and without that medicine your quality of life is going to deteriorate significantly it's not inventing a new drug it's there yes yes well we are going to do what we can with Mr Catalyst and tell them that it is not acceptable that thousands of people are frightened to death about what happens to them yes they are I think you have uh articulated uh exactly what the issue is work together yes please please get the word out to your uh
to people who are suffering from limbs that some of us here are going to do our best thank you all right I will communicate that to them Mr Sanders thank you so much all right Rebecca thanks so much for taking the time thank you take care bye-bye
