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Mashup Score: 0
Step therapy has been a big challenge for my family. Just this year, three of my immediate family members were taken off medications that were working very well and were required to try other, less expensive medications. Why change what is working and unsettle patients’ quality of life? Step therapy should not be used for patients who are already stable on a medication that is effective for them. My seven-year-old daughter, Kinsley, has digestive issues and is no longer able to eat by mouth or accept
Source: rarediseases.orgCategories: General Medicine News, Rare DiseaseTweet
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Mashup Score: 0The Sensations - Let Me In - 13 hour(s) ago
The Sensations – Let Me In, an upbeat number from the swingin early 60’s oldies post- doo wop era. Please enjoy more of the best oldies here: https://www.you…
Source: www.youtube.comCategories: General Medicine News, Rare DiseaseTweet
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Mashup Score: 8
Objective: Amyotrophic lateral sclerosis (ALS) is an incurable, progressive neurodegenerative disease with a significant health burden and poorly understood etiology. This analysis assessed the nar…
Source: www.tandfonline.comCategories: General Medicine News, Rare DiseaseTweet
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Mashup Score: 4Caring for young caregivers, a hidden population - 14 hour(s) ago
Tending to the well-being of loved ones who might fill caregiving roles, especially at vulnerable young ages, is an investment in the well-being of society at large.
Source: www.statnews.comCategories: General Medicine News, Rare DiseaseTweet
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Mashup Score: 0
Approximately 31,000 people are living with amyotrophic lateral sclerosis (ALS) in the United States, with an average of 5,000 new cases every year, according to the Centers for Disease Control and Prevention.
Source: medicalxpress.comCategories: General Medicine News, Rare DiseaseTweet
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Mashup Score: 1
Researchers successfully used a brain-computer interface to synthesize speech directly from brain activity in an ALS patient, achieving 80% word recognition accuracy by listeners, showcasing the interface’s potential for aiding communication in severe speech impairments.
Source: www.news-medical.netCategories: General Medicine News, Rare DiseaseTweet
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Mashup Score: 1Accelerated Approval: Withdrawing Gene Therapies Could Be Easier Than Small Molecules - 15 hour(s) ago
The FDA would only withdraw a rare disease gene therapy for “pretty clear” reasons, such as when there is minimal benefit with extensive side effects. The one-and-done nature of administration also makes withdrawal easier, CBER Director Peter Marks says.
Source: pink.citeline.comCategories: General Medicine News, Rare DiseaseTweet
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Mashup Score: 3Rare Impact Awards - NORD's Rare Impact Awards - 1 day(s) ago
This very special event will celebrate NORD’s legacy and achievements, and honor those individuals and organizations doing remarkable work on behalf of the rare disease community.
Source: rareimpact.orgCategories: General Medicine News, Rare DiseaseTweet-
Meet one of the Policy Changemakers of this year's #RareImpactAwards, Florida @RepGusBilirakis. As Co-Chairman of the #RareDisease Caucus, Rep. Bilirakis advances the understanding that rare diseases are not a rare problem but a shared problem. Read more: https://t.co/7TzeZVz4zT https://t.co/4r3vN9x3T8
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Mashup Score: 1For every million-dollar gene therapy, Peter Marks gets an email from FDA chief Robert Califf - 1 day(s) ago
After Orchard Therapeutics’ gene therapy for a rare genetic disease was approved in March, Peter Marks, M.D., Ph.D., received a simple email from Commissioner Robert Califf, M.D. | After Orchard Therapeutics’ gene therapy for a rare genetic disease was approved in March, Peter Marks, M.D., Ph.D., received a simple email from Commissioner Robert Califf, M.D.
Source: www.fiercepharma.comCategories: General Medicine News, Rare DiseaseTweet
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Mashup Score: 6
Please join Rep. Quigley and the ALS Caucus Co-Chairs, Reps. Calvert, Crow, Fitzpatrick, and Sewell, for an event highlighting research and care for ALS. ALS…
Source: www.youtube.comCategories: General Medicine News, Rare DiseaseTweet
In 2023, #Georgia and #NorthCarolina expanded Medicaid access, enabling more people with #RareDiseases to get care. However, 10 states STILL have not expanded #Medicaid eligibility. Is your state one of them? Find out in the NORD State Report Card, here: https://t.co/H1dnarN9HR https://t.co/a0pzWnQuTp