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Mashup Score: 1Loader - 4 hour(s) ago
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Source: CheckRare.comCategories: General Medicine News, Oncologists1Tweet
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Mashup Score: 1Long-Term Data of Trofinetide in Patients With Rett Syndrome - 8 hour(s) ago
Alan Percy, MD, discusses long-term data from clinical trial extension of trofinetide in patients with Rett syndrome.
Source: CheckRare.comCategories: General Medicine News, Oncologists1Tweet-
Alan Percy, MD, University of Alabama, Birmingham and lead author for the LILAC-1 and LILAC-2 studies, discusses long-term data from clinical trial extension of trofinetide in patients with Rett syndrome. https://t.co/M5owqPbMOd
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Mashup Score: 2Oral Octreotide in Patients With Acromegaly - 8 hour(s) ago
Maria Fleseriu, MD, discusses a post hoc analysis of three phase 3 studies analyzing the use of oral octreotide in patients with acromegaly.
Source: CheckRare.comCategories: General Medicine News, Oncologists1Tweet-
Maria Fleseriu, MD, Director of the Pituitary Center at Oregon Health and Science University, discusses a post hoc analysis of three phase 3 studies analyzing the use of oral octreotide in patients with acromegaly. #CheckRare #RareDisease #Acromegaly https://t.co/mjxLl6T5NC
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Mashup Score: 1Delayed Diagnosis for Prader-Willi Syndrome - 12 hour(s) ago
Evelien Gevers, MD, pediatric endocrinologist at Barts Health and Queen Mary University, discusses the delayed diagnosis for Prader-Willi syndrome.
Source: CheckRare.comCategories: General Medicine News, Oncologists1Tweet-
Evelien Gevers, MD, pediatric endocrinologist at Barts Health and Queen Mary University of London, discusses the delay in diagnosis for Prader-Willi syndrome. #CheckRare #RareDisease #PraderWilliSyndrome https://t.co/syLWeePk4l
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Mashup Score: 4Learn About WHIM Syndrome - 13 hour(s) ago
WHIM syndrome is a rare, congenital primary immunodeficiency disorder associated with severe neutropenia that affects all ages.
Source: CheckRare.comCategories: General Medicine News, Rare DiseaseTweet-
WHIM syndrome is a rare, congenital primary immunodeficiency disorder associated with neutropenia that typically presents in childhood or adolescence Check out our learning page for more information #checkrare #WHIMSyndrome https://t.co/XDxcWgSIEm
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Mashup Score: 1Ataxia Telangiectasia - 1 day(s) ago
Ataxia telangiectasia is a rare primary immunodeficiency that affects the nervous system, the immune system, and other parts of the body.
Source: CheckRare.comCategories: General Medicine News, Oncologists1Tweet-
Ataxia telangiectasia (A-T) is a rare primary immunodeficiency that affects the nervous system, the immune system, and many other parts of the body. To learn more visit our Learning Page at https://t.co/XJtMtjpYu6 #CheckRare #RareDisease #AtaxiaTelangiectasia https://t.co/mBvTAUI7KF
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Mashup Score: 1
Dean Suhr, co-founder and President of the MLD Foundation, discusses the challenges of gaining RUSP approval for MLD.
Source: CheckRare.comCategories: General Medicine News, Onc News and JournalsTweet-
Deah Suhr, co-founder and President of the MLD Foundation, discusses the need to get MLD as part of the Recommended Uniform Screening Panel (RUSP) and the recently approved Lenmeldy (atidarsagene autotemcel) and its effects on families. https://t.co/oHgxeVPgF6
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Mashup Score: 0FDA Grants Rare Pediatric Disease Designation to NS-050/NCNP-03 for the Treatment of Duchenne Muscular Dystrophy - 4 day(s) ago
The U.S. FDA has granted Rare Pediatric Disease Designation to NS-050/NCNP-03 for the treatment of Duchenne muscular dystrophy (DMD).
Source: CheckRare.comCategories: General Medicine News, Onc News and JournalsTweet-
The @US_FDA has granted Rare Pediatric Disease Designation to NS-050/NCNP-03 for the treatment of Duchenne muscular dystrophy. #CheckRare #RareDisease #DMD #FDA https://t.co/0awefqa7n2
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Mashup Score: 1Role of AI in Lysosomal Disorders: Big Data to Artificial Intelligence and Challenges - 4 day(s) ago
Learn about the role of AI in suspecting or diagnosing LDs.
Source: CheckRare.comCategories: General Medicine News, Onc News and JournalsTweet-
CME Program: Pilar Giraldo, MD, Shoshana Revel-Vik, MD, and John L Jefferies, MD discuss the Role of AI in Lysosomal Disorders: Big Data to Artificial Intelligence and Challenges #CheckRare #RareDisease #CME https://t.co/GN1EOXDqvn https://t.co/U2POgyj1xR
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Mashup Score: 2Supplemental Data on the Efficacy of Fenfluramine to Treat Dravet Syndrome-Associated Seizures - 4 day(s) ago
Kelly Knupp, MD, discusses supplemental data on the efficacy of fenfluramine in treating Dravet syndrome-associated seizures.
Source: CheckRare.comCategories: General Medicine News, Oncologists1Tweet-
Kelly Knupp, MD, Associate Professor of Pediatrics and Neurology at the University of Colorado, discusses supplemental data on the efficacy of fenfluramine in treating Dravet syndrome-associated seizures. #CheckRare #RareDisease #DravetSyndrome https://t.co/joei5YP4Qn
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Bruce Leuchter, MD, co-founder and CEO of Neurvati Neurosciences and GRIN Therapeutics, discusses topline results from clinical trial testing investigational radiprodil for GRIN-related disorders. Learn more at: https://t.co/60k4seyh5f #CheckRare #RareDiseases #GRIN