It is no secret that since I became sick and was stuck on prednisone for months that I began to hate my body. After all, I’m up about 30 pounds from where I was nearly two years ago and I lost a lot of muscle that I had worked so hard to gain. I never acknowledged to myself how much I hate my body until I was out for a walk tonight. My hatred isn’t just for the weight gain or loss of muscle. It comes from a feeling of betrayal: my body tried to kill me. My immune system malfunctioned and for some miraculous reason, I survived months of physically challenging work in heels without internally bleeding to death.
I haven’t abandoned this blog, and if you follow me on Twitter or on the Facebook page you have seen my involvement in many projects. This month I have law school exams so I am extremely busy. I will update everyone soon!
I will be spending the day at the National Institutes of Health, checking out the Rare Disease United Foundation’s Beyond the Diagnosis art exhibit, touring the facilities and speaking with researchers. In the evening I’m set to attend a cocktail reception and documentary screening.
Today is my second rare disease day as a patient and completely different from my last one. Now I feel a sense of authority over my disease, the new normal I have created and my spot in working toward change for our community. The game is totally different now, I am not looking to play along, I am working on my own projects.
Wednesday is when I will be on the hill, but at the moment my schedule is uncertain as I have been asked to participate in a few exciting projects. Follow my Instagram and Twitter pages for up-to-the-minute updates on what’s shaking in DC. And if you’re a VC reading this, let’s chat.
If you see me out and about, please come and say hi. My hair will stand out, I promise you can’t miss me (and I don’t bite).
I am headed up to Washington, DC on Sunday for Rare Disease Week. I am excited to be able to meet with a lot of different groups to work on legislation for the 30 million of us fighting these disorders.
Last year was about me, I was focused on what I went through. This year is about YOU, the patient, the caregiver, the healthcare provider, the researcher. Now we have to work on unity between everyone involved in the rare disease space. We need to see to it that the 21st Century Cures bill is signed into law as soon as possible.
I expect this trip to be quite different, as I have found myself at peace with what life has thrown at me.
If you’re going to be in DC and at the events, I would love to meet you. Tweet me @RareCandace and let me know what you’re up to.
I’m alive today because I relied on scientific research to find a treatment for my rare disease. Without the drug I used off-label, I wouldn’t be alive today. My internal bleeding was becoming progressively worse, and it was only a matter of time before I faced a situation that could have had a terrible outcome.
So why am I writing about science? Because today the rare disease community, our families and friends need to unite to send a message to the FDA. Next Friday is an important meeting for the Duchenne Muscular Dystrophy (DMD) community as Sarepta’s Eteplirsen is up for review. This drug has the potential to save many lives.
I have seen first hand via social media just how powerful Eteplirsen is. With DMD, boys are not able to walk once they reach their teenage years. Rare family members have gifted us with videos and photos of their sons who are participating in the Eteplirsen clinical trials walking, playing, and just being normal kids. They’re not losing abilities like the children who aren’t receiving the drug. While it is heartwarming to see improvements, I am constantly reminded that other families are waiting while their children are losing strength. Time is of the essence, Duchenne Muscular Dystrophy is not waiting for FDA approval of Eteplirsen. Just about every week my timeline lights up in remembrance of another son lost. How many more of our boys have to die before the FDA approves Eteplirsen?
There are no safety issues with Eteplirsen, I want to highlight that. The FDA has taken issue with statistical data. Let me explain to you why this enrages me, and why I see this as an attack on our entire rare family, all 30 million of us.
In October 2014, there was no statistical data to tell me that Rituxan would put my ITP in remission. In fact, if I just analyzed raw data, I had less than a 50% chance of it working. Why? Because Rituxan was being used on an entire ITP population, not those who had platelet destruction issues which the CD-20 inhibitor would address. Instead, I used patient data- meaning I analyzed what those who had successful Rituxan treatments had in common. I found the common links on our blood work, how we responded to other treatments, what our doctors thought, even how we bled. I researched the science behind how Rituxan works, what exactly it does to the body and how that relates to the destruction of my platelets. I went in to my doctor and I asked for Rituxan. I knew it was my only hope, I knew my life depended on it. And I was certain that with my research, I had an excellent chance of success.
Rituxan saved my life, but it was NOT a miracle, it was science.
Eteplirsen is to Duchenne Muscular Dystrophy what Rituxan is to Immune Thrombocytopenia. It isn’t about statistics because these treatments aren’t meant for everyone and you cannot apply mass-market measures to orphan drugs for a complete analysis. You cannot expect small patient populations to produce statistic data sets that rival those of drug trials for diseases like diabetes or COPD.
Success for orphan drugs lies in science. It is in the photos and videos of 15 year old Billy (above with Pluto), who still has the ability to walk. This wouldn’t be possible without Eteplirsen.
Billy is living proof that Eteplirsen works. He is the walking scientific evidence of a safe and effective treatment for Duchenne Muscular Dystrophy.
So friends, I ask that you take a few seconds to fill out this short form that will request members of Congress to sign a letter urging the FDA to apply the FDASIA tools to Duchenne which would allow for an accelerated approval of Eteplirsen. This historic moment will set the stage for future orphan drug development, so your participation is critical.
Parent Project Muscular Dystrophy has a short form you fill out with your name and address, and they will send the letter to your representative for you.
Let’s end Duchenne.
Kona wanted to make a special holiday appearance in honor of our call to action for the Senate over the 21st Century Cures initiative. I sincerely hope that when I arrive in DC in a few weeks, progress will have been made. There is no time to waste when 30 million Americans depend on Congress to take action.
My blog is a road map of my rare disease journey, starting last year when I decided to take a chance and use Rituxan off label. It is not approved for ITP patients. Why? Because we don’t have enough information and research on ITP to conduct a comprehensive clinical trial. One of my goals for 2016 is to change that. Through my research, I have been able to isolate a population of ITP patients who can and will benefit from use of the drug. Simply put, this drug is a life saver and the people who achieve remission from it enjoy a few years of a fairly normal life. There is absolutely no reason why this should be denied to people like myself.
Tomorrow is December 18th, which happens to be the one year anniversary of my remission. I truly believe this gift was bestowed upon me to give me a chance to fight for others. I have lost a few friends in the past year to ITP, it doesn’t get any easier and it makes me realize just how fortunate I am.
I’ve spent the last 365 building my new normal, now it is time to change medicine.
Since being diagnosed with fibromyalgia right before my birthday, conserving spoons has become a priority. About this time last year, I wrote a gift guide for prednisone. Now I want to compile a list of gift ideas for people who suffer from chronic illnesses and fatigue. These gifts will help out that spoonie in your life! Continue reading
I promise I haven’t forgotten about my blog. I have just been so busy with a few projects that I haven’t had time to update. Today marks one year since I started RareCandace.com and I still can’t believe 365 days have gone by! Continue reading
I admit I hate my body. I hate that it fights with itself, I hate that is destroys platelets and my joints. I hate the way I look after almost eight months of heavy steroids. I can’t fit into my clothing, I feel disgusting and I’m ashamed of what has happened because of those evil white pills. I lost every sense of my ‘healthy’ self, and the process of getting back to where I use to be is extremely slow. Continue reading
I want to congratulate the following people on winning a signed copy of How To Heal A Bruise!
Thank you to everyone for posting your ITP Awareness Day photos! Remember that awareness and advocacy is important every day of the year.