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Mashup Score: 0WHIM Syndrome - 1 year(s) ago
WHIM syndrome is a rare, congenital primary immunodeficiency disorder associated with neutropenia that typically presents in childhood or adolescence.
Source: CheckRareCategories: Hem/Onc News and Journals, Latest HeadlinesTweet
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Mashup Score: 1
Ari Zimran, MD discusses the history of Gaucher disease research, current treatment options and future research opportunities.
Source: CheckRareCategories: Hem/Onc News and Journals, Latest HeadlinesTweet-
CME: Dr Ari Zimran, Founder and Director of the Gaucher Unit at Shaare Zedek Medical Center, Jerusalem, Israel, Discusses the history of Gaucher disease research, current treatment options and future research opportunities for Gaucher disease. @LDRTC_USA https://t.co/bwDqkbyBKa https://t.co/vuXpXgaqTx
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Mashup Score: 3Primary Mitochondrial Myopathy Explained - 1 year(s) ago
Dr. Alex Dorenbaum, from Reneo Pharmaceuticals, gives an overview of primary mitochondrial myopathies (PMM).
Source: CheckRareCategories: Hem/Onc News and Journals, Latest HeadlinesTweet-
Alex Dorenbaum, MD, Chief Medical Officer of Reneo Pharmaceuticals, explains primary mitochondrial myopathies #PMM #checkrare #rarediseases https://t.co/qQt43s4fRX
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Mashup Score: 0Updated GM1 Gangliosidosis Gene Therapy Trial - 1 year(s) ago
Dr. Samiah Al-Zaidy discusses the updated data from Imagine-1 evaluating PBGM01 in GM1 gangliosidosis presented at WORLDSymposium 2023.
Source: CheckRareCategories: Hem/Onc News and Journals, Latest HeadlinesTweet-
Samiah Al-Zaidy, MD, Vice President of Clinical Development and Lead on the Passage Bio GM1 Gangliosidosis #GM1 Program, discusses the updated data from Imagine-1 evaluating PBGM01 in GM1, which was presented at WORLDSymposium 2023 #checkrare @CureGM1 https://t.co/2f9VspQLcg
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Mashup Score: 0Long Term Efficacy of Enzyme Replacement Therapy (ERT) in Gaucher Disease Type 1 and Type 3 - 1 year(s) ago
Dr. Pramod Mistry discussed the efficacy of enzyme replacement therapy in both Gaucher disease type 1 and type 3.
Source: CheckRareCategories: Hem/Onc News and Journals, Latest HeadlinesTweet-
Pramod Mistry, MD, Ph.D., Professor of Medicine at Yale University School of Medicine, discusses the efficacy of enzyme replacement therapy #ERT in both Gaucher disease type 1 and type 3 #checkrare #rarediseases #gaucherdisease @GaucherNews https://t.co/MII3Z6Zkju
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Mashup Score: 0Metachromatic Leukodystrophy Explained - 1 year(s) ago
Dr. Paul Orchard, from the University of Minnesota Medical School, provides an overview of metachromatic leukodystrophy (MLD).
Source: CheckRareCategories: Hem/Onc News and Journals, Latest HeadlinesTweet-
Paul Orchard, MD, from the University of Minnesota Medical School, provides an overview of metachromatic leukodystrophy #MLD #checkrare #rarediseases @MLDfoundation https://t.co/4CdJFZ1wys
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Mashup Score: 1Targeting SORT1-Positive Cancers - 1 year(s) ago
Dr. Christian Marsolais discusses three studies recently presented at the AACR conference targeting SORT1-positive cancers.
Source: CheckRareCategories: Latest Headlines, Rare DiseaseTweet-
Christian Marsolais, Ph.D., Chief Medical Officer of Theratechnologies, discusses three studies presented at the American Association for Cancer Research #AACR conference targeting SORT1-positive cancers #checkrare #rarediseases @AACR @Theratech_ https://t.co/RuKuGtSP0n
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Mashup Score: 1Rett Syndrome Is a Neurodevelopmental Disorder (and Not a Neurodegenerative Disorder) - 1 year(s) ago
Dr. Smith-Hicks, Neurologist and Director of the Center for Synaptic Disorders at the Kennedy Krieger Institute, gives a detailed overview of Rett syndrome.
Source: CheckRareCategories: Latest Headlines, Rare DiseaseTweet-
Constance Smith-Hicks, MD, Ph.D., Neurologist and Director of the Center for Synaptic Disorders at the Kennedy Krieger Institute, gives a detailed overview of Rett syndrome #checkrare #rarediseases #rettsyndrome @rettsyndnews @Rettsyndrome https://t.co/mmBBayWNZy
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Mashup Score: 0
This is the second orphan drug to be approved for this rare condition.
Source: CheckRareCategories: Hem/Onc News and Journals, Latest HeadlinesTweet-
The U.S. Food and Drug Administration #FDA has approved odevixibat (Bylvay) to treat cholestatic pruritus in patients from 12 months of age with Alagille syndrome #checkrare #rarediseases @rda_algs https://t.co/1geavUFpV6
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Mashup Score: 0Advancements in Cancer Diagnosis - 1 year(s) ago
Dr. Milan Radovich discusses the use of artificial intelligence in advancing the diagnosis of rare cancers and other diseases.
Source: CheckRareCategories: Hem/Onc News and Journals, Latest HeadlinesTweet-
Milan Radovich, PhD, Chief Scientific Officer at Caris Life Sciences, discussed the use of artificial intelligence #AI to improve the diagnosis of different cancer patients #checkrare #rarediseases #cancerdiagnosis @carisls @theNCI https://t.co/zqDO80FKmZ
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Check out our learning page on WHIM Syndrome #checkrare #rarediseases #WHIMSyndrome https://t.co/303s5ZCbAx