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Mashup Score: 1Accelerated Approval: Withdrawing Gene Therapies Could Be Easier Than Small Molecules - 1 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: 1For every million-dollar gene therapy, Peter Marks gets an email from FDA chief Robert Califf - 16 hour(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: 6Working to End ALS: The State of Research and Care in the U.S. - 16 hour(s) ago
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
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Mashup Score: 0Truth Be Told | Notre Dame Magazine | University of Notre Dame - 23 hour(s) ago
The writers who craft ad copy have learned well from poets. But the words they wield have a different intent and power.
Source: magazine.nd.eduCategories: General Medicine News, Rare DiseaseTweet
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Mashup Score: 1
Madras motor neuron disease (MMND) is a rare childhood or juvenile motor neuron disease. Herein, we present a unique case of MMND in an 18-year-old patient, which challenges the conventional und…
Source: www.cureus.comCategories: General Medicine News, Rare DiseaseTweet
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Mashup Score: 1
The following is a summary of “Diagnostic value of neurofilaments in differentiating motor neuron disease from multifocal motor neuropathy,” published in the April 2024 issue of Neurology by Wohnrade et al. Researchers conducted a retrospective study to assess the accuracy of serum neurofilament light chain (NfL) and cerebrospinal fluid (CSF) phosphorylated neurofilament heavy chain (pNfH)
Source: www.physiciansweekly.comCategories: General Medicine News, Rare DiseaseTweet
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Mashup Score: 0Broken String Biosciences and the Francis Crick Institute collaborate to advance ALS research - 1 day(s) ago
Partnership to expand applications of DNA break-mapping technology and advance understanding of genomic instability in development of the neurodegenerative disease amyotrophic lateral sclerosis.
Source: www.news-medical.netCategories: General Medicine News, Rare DiseaseTweet
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Mashup Score: 1
PRESS RELEASE A NEW PUBLICATION IN THE MEDICAL JOURNAL MUSCLE & NERVE VALIDATES THE USE OF THE RATE OF DECLINE OF ALSFRS-R SCORE (ΔFS) FOR THE DESIGN…
Source: www.globenewswire.comCategories: General Medicine News, Rare DiseaseTweet
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Mashup Score: 2
/PRNewswire/ — NeuroSense Therapeutics Ltd. (Nasdaq: NRSN) (“NeuroSense”), a company developing novel treatments for severe neurodegenerative diseases,…
Source: www.prnewswire.comCategories: General Medicine News, Rare DiseaseTweet
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Mashup Score: 2Every 90, 85, 80, 75 Minutes - 2 day(s) ago
“Every 90 Minutes” has been a tagline that organizations have used for over a decade to describe how often someone dies from ALS in the US. …
Source: als-advocacy.blogspot.comCategories: General Medicine News, Rare DiseaseTweet
RT @PharmaPinkSheet: Accelerated Approval: Withdrawing Gene Therapies Could Be Easier Than Small Molecules https://t.co/W5S9H5DaiH #PinkShe…