MD Magazine: Specialty: Rare Disease Report<sup>®</sup> Copyright (c) 2017 Intellisphere, LLC. All rights reserved. http://www.mdmag.com/specialty/rare-diseases/rss Rare Disease Report® en-us Sun, 26 Jan 2020 02:25:31 UTC FDA Approves Teprotumumab for Thyroid Eye Disease https://www.mdmag.com/medical-news/fda-teprotumumab-thyroid-eye-disease Tue, 21 Jan 2020 20:30:00 UTC Targeted Treatment for Gastrointestinal Stromal Tumor Mutation Gains FDA Approval https://www.mdmag.com/medical-news/gastrointestinal-stromal-tumor-mutation-fda Thu, 09 Jan 2020 21:30:00 UTC The 10 Most Read Articles of 2019 https://www.mdmag.com/medical-news/the-10-most-read-articles-2019 Tue, 31 Dec 2019 15:21:00 UTC FDA Expands ALS Oral Drug Riluzole to Feeding Tube Use https://www.mdmag.com/medical-news/fda-als-oral-drug-riluzole-feeding-tube-use Fri, 13 Dec 2019 20:24:00 UTC FDA Approves Golodirsen Injection for DMD with Genetic Mutations https://www.mdmag.com/medical-news/fda-approves-golodirsen-injection-dmd-genetic-mutations Fri, 13 Dec 2019 14:36:00 UTC FDA Authorizes Tool For Duchenne Muscular Dystrophy Screening in Newborns https://www.mdmag.com/medical-news/fda-authorizes-tool-for-duchenne-muscular-dystrophy-screening-in-newborns Thu, 12 Dec 2019 18:25:00 UTC Potential Sickle Cell Cure Targets BCL11A https://www.mdmag.com/conference-coverage/ash-2019/sickle-cell-cure-bcl11a Wed, 11 Dec 2019 15:41:00 UTC ASH Data Points to Correlation Between Biomarkers and Sickle Cell Outcomes https://www.mdmag.com/conference-coverage/ash-2019/ash-biomarkers-sickle-cell-outcomes Tue, 10 Dec 2019 20:04:00 UTC Navitoclax/Ruxolitinib Combination Improves Outcomes for Patients With Myelofibrosis https://www.mdmag.com/conference-coverage/ash-2019/navitoclax-ruxolitinib-combination-improves-outcomes-for-patients-with-myelofibrosis Tue, 10 Dec 2019 19:59:00 UTC Adding Daratumumab to Myeloma Treatment Reduces Risk of Progression, Death https://www.mdmag.com/conference-coverage/ash-2019/daratumumab-myeloma-treatment-reduces-progression-death Tue, 10 Dec 2019 17:58:00 UTC CC-486 Could Represent New Therapeutic Standard for AML Patients https://www.mdmag.com/conference-coverage/ash-2019/cc486-new-therapeutic-standard-acute-myeloid-leukemia-patients Tue, 10 Dec 2019 15:59:00 UTC Sutimlimab Improves Hemoglobin in Cold Agglutinin Disease https://www.mdmag.com/conference-coverage/ash-2019/sutimlimab-improves-hemoglobin-cold-agglutinin-disease Tue, 10 Dec 2019 15:39:00 UTC Blinatumomab Outperforms Chemo in Children, Young Adults with B-ALL https://www.mdmag.com/conference-coverage/ash-2019/blinatumomab-outperforms-chemo-in-children-B-Acute-Lymphoblastic-Leukemia Tue, 10 Dec 2019 13:00:00 UTC Bispecific CAR-T Cell Therapy Shows Promise Treating RRMM https://www.mdmag.com/conference-coverage/ash-2019/bispecific-cart-cell-therapy-rrmm Tue, 10 Dec 2019 01:00:00 UTC Autologous Stem Cell Transplants Are Safe for Older Multiple Myeloma Patients https://www.mdmag.com/conference-coverage/ash-2019/autologous-stem-cell-transplants-safe-older-multiple-myeloma-patients Mon, 09 Dec 2019 22:48:00 UTC Novel Agent Shows Early Promise for Advanced Myelofibrosis https://www.mdmag.com/conference-coverage/ash-2019/novel-agent-shows-early-promise-for-advanced-myelofibrosis Mon, 09 Dec 2019 20:32:00 UTC Young Sickle Cell Adult Patients Experience Increased Fragmented Care https://www.mdmag.com/conference-coverage/ash-2019/young-sickle-cell-adults-increased-fragmented-care Mon, 09 Dec 2019 19:25:00 UTC Interim Analysis Suggest Azacitidine with Enasidenib More Effective Than Monotherapy https://www.mdmag.com/conference-coverage/ash-2019/azacitidine-with-enasidenib-more-effective-than-monotherapy Mon, 09 Dec 2019 17:24:00 UTC Increased AFib, Major Bleeding with Ibrutinib for Hematologic Malignancies https://www.mdmag.com/conference-coverage/ash-2019/increased-afib-major-bleeding-with-ibrutinib-for-hematologic-malignancies Mon, 09 Dec 2019 15:11:00 UTC ASH Study Examines Racial Disparities in AML Clinical Trials https://www.mdmag.com/conference-coverage/ash-2019/ash-racial-aml-trials Sun, 08 Dec 2019 13:00:00 UTC Machine-Learning Model Predicts Treatment Response in Patients with MDS https://www.mdmag.com/conference-coverage/ash-2019/machinelearning-predicts-treatment-response-patients-with-mds Sun, 08 Dec 2019 10:00:00 UTC Mosunetuzumab in Patients with CAR T Refractory non-Hodgkin Lymphoma https://www.mdmag.com/conference-coverage/ash-2019/mosunetuzumab-in-patients-car-t-refractory-non-hodgkin-lymphoma Sat, 07 Dec 2019 13:00:00 UTC Ann Mullally, MD: The Team and Tools for MPNs https://www.mdmag.com/conference-coverage/ash-2019/ann-mullally-team-tools-mpns Fri, 06 Dec 2019 22:43:00 UTC New Recommendations for Sickle Cell Disease Therapies Presented at ASH https://www.mdmag.com/conference-coverage/ash-2019/sickle-cell-disease-therapies-ash Fri, 06 Dec 2019 18:51:00 UTC FDA Approves Voxelotor for Sickle Cell Disease https://www.mdmag.com/medical-news/fda-voxelotor-sickle-cell-disease Mon, 25 Nov 2019 23:11:00 UTC FDA Approves First Acute Hepatic Porphyria Drug https://www.mdmag.com/medical-news/fda-first-acute-hepatic-porphyria-drug Wed, 20 Nov 2019 19:15:00 UTC FDA Approves Crizanlizumab for Sickle Cell Complications https://www.mdmag.com/medical-news/fda-crizanlizumab-sickle-cell-complications Fri, 15 Nov 2019 21:08:00 UTC Mepolizumab Reduces Hypereosinophilic Syndrome Flares https://www.mdmag.com/medical-news/mepolizumab-hypereosinophilic-syndrome-flares Wed, 13 Nov 2019 20:39:00 UTC FDA Approves Lusaptercept-aamt for Anemia in Patients with Beta Thalassemia https://www.mdmag.com/medical-news/fda-approves-lusaptercept-aamt-reblozyl-anemia-beta-thalassemia Fri, 08 Nov 2019 22:58:00 UTC Evan Dellon, MD: Future Treatments for EGID https://www.mdmag.com/conference-coverage/acg-2019/dellon-treatments-egid Tue, 29 Oct 2019 19:00:00 UTC