Playing catch up again for Days 7️⃣ and 8️⃣ in our COUNTDOWN TO MPS AWARENESS DAY!
Day 7: Are there any treatments for MPS/ML to slow down the progression and severity of symptoms?
Although there is no cure for MPS nor ML, five of the syndromes have approved enzyme replacement therapy treatments. Those five syndromes are MPS I, MPS II, MPS IV, MPS VI, and MPS VII. Having treatments make a tremendous difference in quality of life. It’s incredible that within the past 20 years our community has gone from having zero syndromes with any treatment options to presently having FIVE of our syndromes having treatments! That is definitely worth celebrating!
Most encouraging is that any research and related findings for ONE syndrome creates translational breakthroughs for all our syndromes. How? People living with MPS/ML share many of the same issues: respiratory failure, cardiac complications, restricted mobility, GI problems, vision impairment, and for some mild to severe cognitive impairment. So research findings for one syndrome open the doors to helping patients with similar problems manifested in other syndromes. Researchers do not position their findings in a model of zero-sum game and for that we’re thankful to be the recipients of those discovery benefits. 💜🚀💜
To learn more about our current treatments and emerging therapies on the horizon, click here 👉🏽 mpssociety.org/learn-about-mps/mps-treatments/
Day 8 — Anything special going on May 15th?
You betcha! Our Advocacy Committee and other volunteers will be meeting with their lawmakers on Zoom to advocate on for our community! Our advocacy efforts have skyrocketed over the years. People just like YOU have joined forces to share their stories to improve the lives of those affected by MPS/ML.
We advocate both at the State and Federal levels for key legislation. Each year the Society works through our established Speaker Bureau program and takes a contingent to Capitol Hill. As constituents, we encourage our lawmakers to pass legislation directly influencing our access to care and medical resources, increase funding for research, incentive pharmaceutical companies to invest in rare disease drug/treatment breakthroughs, and so much more!
Our volunteer advocates share their stories for many reasons, but beautifully YOUR stories, experiences, and support make a difference in the lives of those with MPS and ML today, and those who will be diagnosed in the future. It’s so much fun that we recently created a Youth Advocacy Committee so that our youngsters can proudly have a voice.
It’s not rocket science. We provide you all the training and support to confidently share your rare disease journey with your lawmakers. Our community is inspiring to all who meet us, we will continue to share our heartwarming journeys and create a catalyst for change. 💜🚀💜
To learn more about how you can become an advocate, click here 👉🏽 mpssociety.org/advocacy-for-mps-and-ml/
#BlastOffForACure #MPSAwareness #MPSML #BetterTogether #TheSkyIsTheLimit #CureSupportAdvocate
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Join Courageous Parents Network for a thought-provoking discussion with Jessica Fein, author of Breath Taking: A Memoir of Family, Dreams and Broken Genes. This poignant memoir takes readers on a journey through the roller coaster of complex parenting, exploring themes of love, resilience, and the profound impact of a rare degenerative disease on one family’s existence. Learn more and register for the May 14th conversation at: courageousparentsnetwork.org/events/book-talk-with-jessica-fein-breath-taking-rare-girl-in-a-worl...In her book Breath Taking, Jessica Fein boldly admits that a medical emergency with her daughter Dalia found her making promises, lots of them! Have you had a similar experience?
Hear more from Jessica at her talk with CPN on May 14th
Register: bit.ly/3y1W4IB
#WritingAsHealing #Caregiver #RareDisease #MitoAction #BreathTaking #Memoire #LoveandLoss #GriefJourney #MitoWarrior
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Day 6: How has the National MPS Society pushed for newborn screenings?
The earlier a child gets diagnosed with MPS/ML, the sooner interventions and resources can be deployed to assist a family, minimize irreparable damage to the body as the syndromes progress, and emotionally prepare for the journey ahead. Early diagnosis is paramount to realizing the best outcomes. The National MPS Society led the charge by submitting the official application and pushing for MPS to be added to newborn screening tests for early diagnosis (both federally and individually by state).
On this map you will see the tremendous results of our hard work in adding MPS I, MPS II, and MPS IV to screen newborns in their respective states. We thank our staff for their diligence in keeping our lawmakers accountable and for the volunteers in our community who have assisted in being the voices for vital newborn screening expansion. For every story we share, we can press our lawmakers to turn this map PURPLE! 💜🚀💜
#blastoffforacure #MPSAwareness #MPSML #bettertogether #theskyisthelimit #CureSupportAdvocate
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