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Mashup Score: 0Treatment With NALL Beneficial for Niemann-Pick Disease - 20 hour(s) ago
Treatment with NALL for 12 weeks yields better neurological status than placebo among patients with Niemann-Pick disease type C.
Source: CheckRare.comCategories: General Medicine News, Rare DiseaseTweet
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Mashup Score: 1Rare Disease Impact on Understanding Common Diseases - 20 hour(s) ago
Cat Lutz, PhD, MBA, VP at JAX Rare Disease Translational Center, discusses rare disease research’s impact on understanding common diseases.
Source: CheckRare.comCategories: General Medicine News, Rare DiseaseTweet
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Mashup Score: 1Personalizing Pharmaceutical Care Through AI - 24 hour(s) ago
Bill Grambley explains how his company attacks health inequalities by providing personalized pharmaceutical care with an AI based platform.
Source: CheckRare.comCategories: General Medicine News, Onc News and JournalsTweet
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Mashup Score: 1Loader - 24 hour(s) ago
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Source: CheckRare.comCategories: General Medicine News, Onc News and JournalsTweet
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Mashup Score: 0Loader - 24 hour(s) ago
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Source: CheckRare.comCategories: General Medicine News, Rare DiseaseTweet
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Mashup Score: 1LDRTC Symposium: Newborn Screening and Lysosomal Disorders - 2 day(s) ago
This symposium, led by Ozlem Goker-Alpan, MD of the Lysosomal & Rare Disorders Research & Treatment Center (LDRTC) reviews the current state of newborn screening (NBS) for lysosomal disorders and the need for newer guidelines to address the many unmet needs.
Source: CheckRare.comCategories: General Medicine News, Onc News and JournalsTweet
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Mashup Score: 2Rett Syndrome - 2 day(s) ago
Rett syndrome is a multisystem disorder that primarily affects girls; multiple loss-of-function mutations to the MECP2 gene are the cause of this rare disease.
Source: CheckRare.comCategories: General Medicine News, Onc News and JournalsTweet
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Mashup Score: 2Gene Therapy For Hemophilia B - 2 day(s) ago
Brahm Goldstein, MD, Vice President, R&D, Hematology at CSL Behring, discusses HEMGENIX, a pivotal gene therapy for hemophilia B.
Source: CheckRare.comCategories: General Medicine News, Onc News and JournalsTweet
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Mashup Score: 1Loader - 2 day(s) ago
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Source: CheckRare.comCategories: General Medicine News, Onc News and JournalsTweet
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Mashup Score: 0Loader - 2 day(s) ago
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Source: CheckRare.comCategories: General Medicine News, Onc News and JournalsTweet
Treatment with N-acetyl-L-leucine #NALL for 12 weeks yields better neurological status than placebo among patients with genetically confirmed Niemann-Pick disease type C #CheckRare #RareDisease #NiemannPickTypeC https://t.co/WwHQk46vAu