Health Highlight: Wed. 15, Mar.
Today’s highlights: The FDA has approved medication to help treat symptoms of Rett syndrome; It’s MS Awareness Week (Mar. 12–18).
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FDA Approval for Daybue (trofinetide) to treat Rett Syndrome
✍🏼 By: It’s Technical | Carlos Franco • 🗣️@FDA_Drug_Info, @US_FDA, @AcadiaPharma
Summary: The oral solution form of trofinetide, marketed as Daybue (pronounced: day-BYOO), has received approval from the FDA as the first treatment for the rare genetic disorder Rett syndrome. Daybue has shown improvement in reducing the symptoms of Rett syndrome but also carries a risk of side effects. (1, 2)
What is Rett syndrome? Rett Syndrome is a rare genetic neurological disorder that mainly affects females (about one out of every 10,000 female births worldwide).
- It is caused by a gene mutation in the X chromosome and is typically diagnosed in children aged 6 to 18 months as they begin to miss developmental milestones or lose previously acquired abilities.
- Rett syndrome causes a varying range of disabilities depending on the characteristics of the X chromosome mutation. (3)
What does Daybue do? Acadia Pharmaceuticals Inc., the pharmaceutical company that sought the approval, claims that Daybue has shown greater symptom relief than placebo in those treated with the drug.
“During a 12-week clinical trial of 187 females ages 5 to 20, treatment with DAYBUE demonstrated improvements in the signs and symptoms of Rett syndrome when compared to placebo. The following signs and symptoms were assessed by the RSBQ scale in the clinical trial: breathing, hand movements or stereotypies, repetitive behaviors, night-time behaviors, vocalizations, facial expressions, eye gaze, and mood.” — https://daybue.com/daybue-faq.pdf
What is the takeaway? This approval will hopefully provide another avenue of care for those affected with Rett syndrome, who have a greater than 50 percent chance of reaching the age of 50. (4)
- About Rett Syndrome
- DAYBUE™ (trofinetide) The only FDA-approved treatment for Rett syndrome
- FDA Roundup: March 14, 2023
🏷️ Tags: #FDA #NewDrugApproval #Daybue #AcadiaPharma #RettSyndrome
March 12th to 18th is Multiple Sclerosis (MS) Awareness Week
✍🏼 By: It’s Technical | Carlos Franco • 🗣️ @mssociety, @MSIntFederation, @WorldMSDay
What is Multiple Sclerosis? Multiple sclerosis, or MS, is a chronic neurological disease that affects the tissue of the nervous system — specifically the brain and spinal cord. It affects females more than males and is suspected to be an autoimmune disorder, but its cause is so far unknown. A series of tests and assessments are required to diagnose MS, and treatments are aimed at slowing progression and managing symptoms. (5, 6)
It is important to raise awareness of MS because the number of people in the US diagnosed with the disease has increased dramatically in the past 40+ years. This means you may already know someone who is dealing with this disease and can be a voice for and with them.
“A study by leading experts estimates that in 2017, nearly 1 million adults (up to 913,925) were living with MS in the United States. This is more than twice the previously reported number from a national study in 1975 and subsequent updates.” — https://www.nationalmssociety.org
Is MS Awareness Week the same as World MS Day? The short answer is, no. World MS Day is on May 30th, and it is headed by the Multiple Sclerosis International Federation (MSIF). While the goal is the same, World MS Day does not occur during MS Awareness Week.
“World MS Day is the MS movement’s annual campaign to raise awareness of the condition, and to support and connect the 2.8 million people living with it worldwide. It is a day to celebrate global solidarity and hope for the future.” — https://www.msif.org/
So what can I do to help? According to the National MS Society, you can, “Share your story or connect with a person affected by MS… Follow the Society’s social media channels and share with your networks to help ensure that more people understand what life with MS can be like and engage with them to take action.” (7)
Personally, I’ve had at least three people in my life diagnosed with MS; a teacher, a coworker, and a friend. It’s important to get the message out there that…
You are seen. You are important. We support you.
- Multiple Sclerosis (MS)
- Consumer Health: Are you at risk for MS?
- Where can I find more information about multiple sclerosis?
- World MS Day
🏷️ Tags: #MultipleSclerosis #MS #MSAwarenessWeek #WorldMSDay #MSConnections
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- Accessed on March 15, 2023. (https://www.fda.gov/news-events/press-announcements/fda-roundup-march-14–2023) ↩︎
- Accessed on March 15, 2023. (https://daybue.com/) ↩︎
- Accessed on March 15, 2023. (https://www.rettsyndrome.org/about-rett-syndrome/what-is-rett-syndrome/) ↩︎
- Accessed on March 15, 2023. (https://www.rettsyndrome.org/about-rett-syndrome/faqs/) ↩︎
- Accessed on March 15, 2023. (https://medlineplus.gov/multiplesclerosis.html) ↩︎
- Accessed on March 15, 2023. (ninds.nih.gov/health-information/disorders/multiple-sclerosis) ↩︎
- Accessed on March 15, 2023. (https://www.nationalmssociety.org/Get-Involved/Raise-Awareness#section-1) ↩︎