MD Magazine: Specialty: Rare Diseases Copyright (c) 2017 Intellisphere, LLC. All rights reserved. http://www.mdmag.com/specialty/rare-diseases/rss Rare Diseases en-us Tue, 23 Apr 2019 09:13:50 UTC FDA Accepts NDA for Riluzole Oral Film for ALS Treatment http://www.mdmag.com/medical-news/fda-accepts-nda-riluzole-oral-film-als-treatment Tue, 16 Apr 2019 16:13:00 UTC ALS Gene Expression Atlas Could Provide New Disease Insights http://www.mdmag.com/medical-news/als-gene-expression-atlas-could-provide-new-disease-insights Fri, 05 Apr 2019 13:00:00 UTC Benralizumab as a Treatment for Hypereosinophilic Syndromes http://www.mdmag.com/medical-news/benralizumab-as-a-treatment-for-hypereosinophilic-syndromes Fri, 05 Apr 2019 11:00:00 UTC Luxturna Successfully Used to Treat Inherited Retinal Disease http://www.mdmag.com/medical-news/luxturna-treat-inherited-retinal-disease Sat, 30 Mar 2019 09:00:00 UTC FDA Designates PRM-151 Breakthrough Therapy for Idiopathic Pulmonary Fibrosis http://www.mdmag.com/medical-news/fda-prm151-breakthrough-therapy-idiopathic-pulmonary-fibrosis Tue, 26 Mar 2019 14:03:00 UTC Biallelic Mutations Causing Rare Pediatric Brain Disorder VARS http://www.mdmag.com/medical-news/biallelic-mutations-causing-rare-pediatric-brain-disorder-vars Tue, 12 Mar 2019 19:52:00 UTC Insights on Rare MICPCH Syndrome Could Help with Autism, Epilepsy http://www.mdmag.com/medical-news/insights-rare-micpch-syndrome-could-help-autism-epilepsy Thu, 07 Mar 2019 18:29:00 UTC FDA Grants Priority Review to 2 Cancer Investigational Drugs http://www.mdmag.com/medical-news/fda-grants-priority-review-to-2-cancer-investigational-drugs Wed, 06 Mar 2019 14:00:00 UTC Age, Sex Are Risk Factors for Arrhythmic Events in Long QT Syndrome http://www.mdmag.com/medical-news/age-sex-risk-factors-arrhythmic-events-long-qt-syndrome Wed, 20 Feb 2019 16:00:00 UTC Experimental ALS Therapy Uses Antibodies to Target TDP-43 protein http://www.mdmag.com/medical-news/experimental-als-therapy-uses-antibodies-to-target-tdp43-protein Mon, 18 Feb 2019 16:02:00 UTC New Therapeutic Target for Myeloproliferative Neoplasm http://www.mdmag.com/medical-news/new-therapeutic-target-myeloproliferative-neoplasm Wed, 13 Feb 2019 17:47:00 UTC FDA Approves Split Infusion for Daratumumab for Multiple Myeloma http://www.mdmag.com/medical-news/fda-approves-split-infusion-daratumumab-multiple-myeloma Tue, 12 Feb 2019 15:34:00 UTC Focus on Therapeutic Target for Butterfly Syndrome http://www.mdmag.com/medical-news/focus-on-therapeutic-target-for-butterfly-syndrome Mon, 11 Feb 2019 17:30:00 UTC Targeting an Immune Regulating Protein May Help Halt Glioblastoma Progression http://www.mdmag.com/medical-news/targeting-immune-regulating-protein-may-help-halt-glioblastoma-progression Fri, 08 Feb 2019 15:35:00 UTC FDA Approves Cablivi for aTTP http://www.mdmag.com/medical-news/fda-approves-cablivi-attp Wed, 06 Feb 2019 18:40:00 UTC Depression Drug Shows Promise as ALS Treatment on Genetic Level http://www.mdmag.com/medical-news/depression-drug-shows-promise-als-treatment-genetic-level Wed, 06 Feb 2019 16:00:00 UTC Engineered Cancer Antivirus Has Antisarcoma Effect http://www.mdmag.com/medical-news/engineered-cancer-antivirus-antisarcoma-effect Mon, 04 Feb 2019 14:45:00 UTC Finding a Path Forward: The Continued Unmet Medical Need in FCS http://www.mdmag.com/medical-news/finding-path-forward-continued-unmet-medical-need-fcs Wed, 30 Jan 2019 16:58:00 UTC Therapeutic Target for Medulloblastoma Identified in Mice Models http://www.mdmag.com/medical-news/therapeutic-target-medulloblastoma-identified-mice-models Tue, 29 Jan 2019 14:46:00 UTC FDA Approves Ibrutinib/Obinutuzumab for Treatment-Naive Patients with Chronic Lymphocytic Leukemia http://www.mdmag.com/medical-news/fda-approves-ibrutinibobinutuzumab-treatmentnaive-patients-chronic-lymphocytic-leukemia Mon, 28 Jan 2019 17:00:00 UTC Understanding a Rare Cancer in Duchenne Muscular Dystrophy Patients http://www.mdmag.com/medical-news/understanding-rare-cancer-duchenne-muscular-dystrophy-patients Fri, 25 Jan 2019 15:00:00 UTC Refining Leukemia Classification for More Accurate Diagnoses http://www.mdmag.com/medical-news/refining-leukemia-classification-more-accurate-diagnoses Wed, 23 Jan 2019 16:30:00 UTC Acute Flaccid Myelitis Paralysis Reversed in New Surgery http://www.mdmag.com/medical-news/acute-flaccid-myelitis-paralysis-reversed-new-surgery Mon, 21 Jan 2019 18:15:00 UTC FDA Approves Trastuzumab for Metastatic Gastric/Gastroesophageal Junction Adenocarcinoma http://www.mdmag.com/medical-news/fda-approves-trastuzumab-metastatic-gastricgastroesophageal-junction-adenocarcinoma Mon, 21 Jan 2019 17:16:00 UTC New Drug to Treat 2 Neglected Tropical Diseases http://www.mdmag.com/medical-news/new-drug-treat-2-neglected-tropical-diseases Fri, 18 Jan 2019 18:12:00 UTC Muscular Dystrophy Treatment with CRISPR Technology on the Way http://www.mdmag.com/medical-news/muscular-dystrophy-treatment-crispr-technology Wed, 16 Jan 2019 19:36:00 UTC FDA Approves Hepatocellular Carcinoma Therapy, Cabozantinib http://www.mdmag.com/medical-news/fda-approves-hepatocellular-carcinoma-therapy-cabozantinib Tue, 15 Jan 2019 16:48:00 UTC Senescent Cell Research Shows Potential for Idiopathic Pulmonary Fibrosis http://www.mdmag.com/medical-news/senescent-cell-research-shows-potential-idiopathic-pulmonary-fibrosis Mon, 14 Jan 2019 18:31:00 UTC Dipanjan Basu, PhD, Highlights Potential of Omipalisib for Neurocutaneous Melanocytosis http://www.mdmag.com/medical-news/dipanjan-basu-phd-highlights-potential-omipalisib-neurocutaneous-melanocytosis Fri, 11 Jan 2019 15:45:00 UTC Chemotherapy Could Elevate Risk for Rare Blood Cancer http://www.mdmag.com/medical-news/chemotherapy-could-elevate-risk-rare-blood-cancer Fri, 04 Jan 2019 16:26:00 UTC