HCPLive<sup>®</sup>: 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 Thu, 02 Apr 2020 21:58:11 UTC FDA Approves Treatment for Patients with Hemophilia with Inhibitors https://www.mdmag.com/medical-news/fda-approves-treatment-patients-hemophilia-inhibitors Wed, 01 Apr 2020 17:53:00 UTC FDA Grants Orphan Drug Designation for Phenylketonuria Treatment https://www.mdmag.com/medical-news/fda-orphan-drug-designation-phenylketonuria-treatment Thu, 26 Mar 2020 15:51:00 UTC Avapritinib Shows Promise in Systematic Mastocytosis Trial https://www.mdmag.com/conference-coverage/aaaai-2020/avapritinib-systematic-mastocytosis-trial Fri, 20 Mar 2020 16:30:00 UTC FDA Approves Osilodrostat, A Non-Surgical Option for Cushing's Disease https://www.mdmag.com/medical-news/isturisa-fda-approves-osilodrostat-nonsurgical-option-cushings-disease-novartis Sat, 07 Mar 2020 13:33:00 UTC Isatuximab-irfc Receives FDA Approval for Previously Treated Multiple Myeloma https://www.mdmag.com/medical-news/isatuximab-irfc-fda-approval-previously-treated-multiple-myeloma Mon, 02 Mar 2020 18:24:00 UTC DocTalk Podcast: Rare Disease Day 2020 https://www.mdmag.com/medical-news/rare-disease-day-2020-podcast Sat, 29 Feb 2020 16:00:00 UTC Rare Genomics Institute Launches Free Rare Disease Device Program https://www.mdmag.com/medical-news/rare-genomics-institute-free-rare-disease-device-program Sat, 29 Feb 2020 15:36:00 UTC Ultrasounds Help Identify Advanced Cystic Fibrosis Liver Disease https://www.mdmag.com/medical-news/ultrasounds-cystic-fibrosis-liver-disease Sat, 29 Feb 2020 14:00:00 UTC Medicare Part D Bills Could Harm Rare Disease Drug Development https://www.mdmag.com/medical-news/medicare-part-d-bills-could-harm-rare-disease-drug-development Sat, 29 Feb 2020 12:00:00 UTC Alkaptonuria Treatment Bettered by Machine Learning Strategy https://www.mdmag.com/medical-news/alkaptonuria-treatment-bettered-by-machine-learning-strategy Sat, 29 Feb 2020 10:00:00 UTC Jamak Modaresi Esfeh, MD: Finding Hepatic Encephalopathy https://www.mdmag.com/medical-news/jamak-modaresi-esfeh-md-finding-hepatic-encephalopathy Wed, 26 Feb 2020 15:29:00 UTC Inovio Files Application for RRP Treatment Trial https://www.mdmag.com/medical-news/INO-3107-fda-approves-trial-recurrent-respiratory-papillomatosis Mon, 10 Feb 2020 17:35:00 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