Action Alert: Ask Congress to Protect the Orphan Drug Tax Credit

Action Alert: Ask Congress to Protect the Orphan Drug Tax Credit

The MSA Coalition respects the views of all members of our groups. Given the relevance of the following issue to the MSA community, we are sharing this important information. Individuals are encouraged to research facts and act accordingly based on personal beliefs in consideration of action on this item.

The MSA Coalition recently signed on to letters sent by the National Organization for Rare Disorders (NORD) to House and Senate leaders opposing proposed changes to the Orphan Drug Tax Credit. The letters urge Congressional leaders to oppose the changes to the Orphan Drug Tax Credit (ODTC) that were included in the House Ways and Means Committee’s portion of the Build Back Better Act. If included, the new provision would negatively impact the ability of rare disease patients to obtain an FDA approved drug that has been proven to be safe and effective for their specific condition.

Specifically, the Ways and Means portion of the Build Back Better Act includes a provision that would curtail the Orphan Drug Tax Credit for qualified clinical testing expenses by removing this critical incentive for all but the first approved orphan use of a drug. The tax credit would therefore be available for only the first approved orphan use of a new drug, and not for any subsequent use, thus diminishing research into rare disease uses. Over 90% of rare diseases lack an FDA-approved treatment indicated for the specific rare disease. Each time a new orphan use of a drug is added to the label of a drug, more rare disease patients receive assurance that the drug is safe and effective for them.

Here are links to the NORD letters:

Senate Letter: http://www.multiplesystematrophy.org/…/ODTC-Letter…

House Letter: http://www.multiplesystematrophy.org/…/ODTC-Letter…

Here is the NORD link that will help you contact your members of Congress if you wish to do so:

https://rareaction.org/take-action/#/127

Please remember that everyone has different views, and any and all comments should be careful to maintain respect for the views of others.

Thankful for Another Day to Fight

Thankful for Another Day to Fight

By Betty Martin

My name is Betty Martin. I have lived with my husband, Jerry Martin – for nearly fifty or sixty years now – in Lynnville, Tennessee.

I had Parkinson’s Disease, but then I was faced with Multiple System Atrophy (MSA). I’ve had it now for about ten years. Each day is a struggle, but with the help of my husband and daughter, Christy, I’m able to meet each day with a new challenge.

As I ponder over the last thirteen years, there have been many ups and downs.

In 2003, I was sitting at my desk, and my right forefinger started jumping. I passed out and off I went to the hospital. I got out of work that day.

I was told that I had “POTS” which stands for Postural Orthostatic Tachycardia Syndrome – what a word – which controls your heart rate and blood flow.

Three months later, I was told that I had Parkinson’s. Not getting any better, I went back to the doctor. Three doctors confirmed I have Multiple System Atrophy (MSA) also known as Shy-Drager Syndrome. That did it. I sat in the doctor’s office and cried.

“Why me, Lord?” I asked.

But why not me? I’m no better than anyone else who has MSA. I just have to fight a little harder. I was not ready to give up, but my battle was not over.

In October 2014, I was faced with more bad news: breast cancer. With the help of great doctors and all my family and friends, they pulled me through hardships once again.

It’s not been easy. Each day is a struggle going in and out of the hospital with fainting spells. Sometimes my feet just simply don’t want to move. I walk with a cane. I can’t run or jump rope, but I have a lot to gain.

On December 27, 2020, I was diagnosed with COVID and Pneumonia and back in the hospital I went.

It was “touch and go” for awhile with so much against me, but the Lord is still not through with me.

“You’ve come so far. Don’t give up now,” were the words that got me through.

With love in my heart, I’ll pray for you, and you pray for me. We’ll beat this disease, and one day, there will be a cure for us.

So, never give up. Let’s fight and live and thank our Lord for helping each of us.

Each morning, I walk outside to look at the flowers, and yes, listen to the birds singing in the trees.

With gladness in my heart, I thank my Lord for hearing my cry, and for giving me another day to fight, to live, and to help someone else along the way.

 

 

A Journey Together

A Journey Together

By Cathy Chapman, Patient Representative, the MSA Coalition

The MSA Coalition Annual Patient & Family Conference is coming up on September 30 – October 2. Reflecting back to the time I was diagnosed in 2012, I was overwhelmed by the fear of the unknown and where to turn for guidance, knowledge, and support. For my family, it was grasping for an understanding of multiple system atrophy, and for others, it meant denial.

After having attended these conferences in person and virtually since 2016, I have found compassion, knowledge from the top medical professionals, and supportive and understanding friendships. For my family, not only did they gain clarity on what I was facing, they found guidance and support as care partners.

We are not alone but part of the MSA family on this journey together.

The MSA Coalition Annual Patient & Family Conference is Sept 30-Oct 2. Register here for free.

 

 

COVID Information for the Multiple System Atrophy Community

COVID Information for the Multiple System Atrophy Community

Updated 8/13/2021: In consideration of the FDA announcement regarding the third vaccine shot, MSA patients are generally not considered to be immunosuppressed. Every case is different however, and the decision to receive a booster shot should be discussed with the clinical team caring for the patient.

While MSA patients as a whole are not considered to be more likely to contract COVID, the consequences of becoming ill with the virus may be more extreme. Vaccination and booster shots should be discussed with the clinical team caring for the patient in order to arrive at the best decision for each patient. Patients who have respiratory compromise would be expected to be more vulnerable because COVID-19 is primarily a disease of the lungs and respiratory tract. Many patients with MSA do develop impaired respiratory (breathing) control and, in more advanced stages, develop pneumonias. As such, we would urge patients with MSA to take especially vigorous precautions against exposure to COVID-19, including frequent hand washing/sanitizing, restricted exposure to others and not delay in seeking medical advice if symptoms of fever, cough or shortness of breath develop.

About The Coronavirus:

COVID-19 (coronavirus disease 2019) is a respiratory tract infection caused by SARS-CoV-2 virus. Coronaviruses are responsible for about 15-20% of common colds, but there are times when new viruses enter the human population for the first time. When this happens, sometimes there is little to no protection from our previous exposures to coronavirus because the virus is so different. This seems to be the case with COVID-19.

The Infection

COVID-19 has been seen mostly in Asia, primarily China, South Korea, and Japan, but has recently spread to Europe (Italy), the middle East (Iran), and the United States with 44 states now reporting cases. While many have experienced only mild symptoms (runny nose, cough, and cold-like symptoms), some people, particularly the elderly and those with underlying medical problems, have experienced severe disease in the lower parts of their lungs (pneumonia). Young adults have been infrequently affected, and children <12 years old have largely been protected from developing COVID-19 disease.

General Concerns

There are two primary concerns that physicians and scientists have raised. First, the mortality rate of COVID-19 may be substantially higher than for other respiratory viruses. Right now, the mortality rate is estimated to be between 1-3%; in contrast, the mortality rate for influenza is about 10-30 times less (about 0.1%). Undoubtedly, the mortality rate will be less than currently estimated since we don’t know the true number of mild infections; nevertheless, there remains a real concern that COVID-19 is more severe than what we are used to seeing with other seasonal viruses. Second, the virus spreads efficiently, being transmitted before symptoms occur and staying in the nose and saliva for days to weeks after symptoms improve.

COVID-19 Misconceptions

There have been numerous misconceptions that have arisen.

The biggest misconception is that masks are not useful for slowing the spread of COVID-19..

Masks are essential for slowing the spread between people:

  • If a person is sick (even asymptomatic) wearing a mask will help keep them from spreading the virus by trapping their air particles in their mask.
  • Masks are now believed to help protect individuals wearing them from getting COVID-19.

Conclusion: You should wear a mask and you should avoid people who are not wearing a mask.

Recommendations for the MSA Community

This is an important time to be prepared but not panicked. Practical steps should be taken by MSA patients and those in close contact with them to lower the risk of exposure to COVID-19:

  • Use careful hand hygiene by washing hands frequently with soap and water or hand sanitizer that contains at least 60% alcohol. In other words, wash your hands like you just touched hot peppers and need to take your contact lenses out.
  • Wear a mask and stay physically distanced (at least 6 feet apart) if you have visitors or must go out.
  • Require any visitor (home nurses, etc.) to wear a mask when in your home.
  • Stay home as much as possible and avoid crowds of people where the risk of spreading and thus getting COVID-19 is high.

These important strategies can help lower your chances of contracting COVID-19.

For the most up to date information on COVID-19 please reference the CDC website.

Watch the MSA Coalition’s Webinar

 

MSA Care Amidst The Pandemic:

COVID-19

Recorded on April 20, 2020

Resources on MSA and COVID-19

The Dysautonomia Center at NYU issued this guidance for MSA patients, including answers to frequently asked questions. This article is an especially useful compendium and we highly recommend it. If you are wondering whether to keep your appointments, or how to handle home health care visits, this is a great resource.

General COVID-19 Information
In the United States, we rely on the Centers for Disease Control and Prevention for information and advice; the website is here.

Information for those enrolled in the Verdiperstat Phase 3 Clinical Trial
Biohaven Pharmaceuticals have provided some answers to patient questions regarding the ongoing administration of this important clinical trial during this time of uncertainty. Read our blog with Q&A here.

Self-care in a distressing time
Living in the time of COVID-19, and especially when also dealing with a difficult disease, requires great psychological energy. Many mental health professionals have good advice for all of us as we practice social distancing, sheltering in place, or, in some cases, are quarantined. Here are a few good practices:

  • Stick to a routine
  • Get outdoors, if you are able, at least once a day for thirty minutes, maintaining social distancing guidelines
  • Stay hydrated and eat good food
  • Get your COVID-19 information from reliable medical sources
  • Try to focus on the good in the world, the people who are helping
  • Remind yourself that this is temporary
  • As much as possible, be kind to yourself and patient with those near and dear to you

If you need help, reach out to appropriate resources and providers in your community. And again, we at the MSA Coalition are a phone call away at 866-737-5999. We will return your call and try to direct you to assistance.
If you would rather use email, our address is info@multiplesystematrophy.org. You can see updated information at our website, www.msacoalition.org.

Communicating with Others
The Coalition maintains several Facebook groups to provide those in the community with information and a place to ask questions. These groups and pages are monitored, and include the following:

The MSA Coalition Facebook Page

Public Groups:

MSA Coalition Public Discussion group (7500 members)
MSA Research News group (2900 members)

Private Groups:

MSA Buddies group (4300 members)
MSA A Patients Journey group (400 members)
MSA Widows and Widowers group (200 members)
Children of MSA Warriors (270 members)
MSA Coalition Volunteers (700 members)

US Regional Groups (Private):

MSA New England regional group
MSA Middle Atlantic regional group
MSA South Atlantic regional group
MSA Northeast central regional group
MSA Northwest central regional group
MSA Southeast central regional group
MSA Southwest central regional group
MSA Mountain regional group
MSA Pacific regional group

We are here to help!
This is uncharted territory for all of us. It will take all of us acting together for the common good to flatten the curve and get through it. History shows us other generations have faced crises and emerged stronger, and we all know that the MSA community shows its resilience time and time again. Together, we are strong.

Stay safe. Take care of each other. Wash your hands.

Practice Saying “Your Choice!”

Practice Saying “Your Choice!”

By Elaine Douglas, The MSA Coalition Board of Directors

Dignity is one of the most important aspects of being human. Simply, dignity is about being seen, heard, and acknowledged for who we are and treated as if we mattered. The realities of having MSA can threaten a person’s sense of dignity. As care partners, we have a key role in helping our loved one maintain their sense of dignity.

This role is important throughout the MSA journey. But there is no more critical time for focusing on this role than at the end of life. One article from a hospice agency explains that “[n]ear the end-of-life, most people have less control over their life due to illness. Therefore, caregivers must act in ways that help preserve the person’s sense of dignity.”

The article goes on to identify key aspects of dignity at this part of the journey. These are:

  • Respect, which includes self-respect, mutual respect, and respect for privacy.
  • Autonomy, which involves having and providing choices, as well as competence and independence.
  • Empowerment, which can involve self-esteem, pride, and modesty.
  • Communication, such as explaining and understanding information, both verbally and non-verbally.

Choice is a critical component of this phase in our journey. Do they want a feeding tube? A tracheostomy? DNR? When would they consider palliative care? Hospice care? And as death nears, who do they want with them? Are they considering Death with Dignity? The process of finalizing advanced directives can help direct your discussions about these decisions. Most importantly, it’s a way for you to learn what they want.

What if you disagree with their choices? You can have open and authentic discussions … maybe many of them. You can share research, information, stories, and feelings. But ethically and legally, patients have the right to make their own healthcare decisions. So, listen and learn; then honor their wishes.

Honoring our loved one’s choices about what they want – and how they die – is the ultimate expression of love and respect.

Music Therapy for MSA

Music Therapy for MSA

By Cathy Chapman, Patient Representative, the MSA Coalition

Music engages multiple areas in the brain involved in emotions, cognition and motor function.

It has been shown that Singing Music Therapy can improve breathing control and exercise vocal muscles that affect swallowing and speech. You can sing along to a recording of your favorite song, or if you need help with the lyrics, search up a “lyric video” or “karaoke video” on YouTube. Have fun with your favorite songs while you exercise your vocal muscles!

Also, creating your own personal collection of songs that bring joy, relaxation and special memories is another way to start your music therapy. Sharing your songs with your care partner, family and friends is a good way to spend time together and start your own Sing-A-Long. There is power in music.