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 Thu, 21 Nov 2019 02:12:47 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 Moira Gunn, PhD: Challenges of Developing Rare Disease Therapies https://www.mdmag.com/conference-coverage/nord-2019/moira-gunn-phd-challenges-developing-rare-disease-therapies Fri, 25 Oct 2019 13:52:00 UTC Dawn Rotellini: Impact of Policy Changes on Patients https://www.mdmag.com/conference-coverage/nord-2019/impact-policy-changes-patients Thu, 24 Oct 2019 20:14:00 UTC Approvals for Rare Diseases Reached Historic High in 2018 https://www.mdmag.com/conference-coverage/nord-2019/approvals-rare-diseases-reached-historic-high-2018 Thu, 24 Oct 2019 16:53:00 UTC Dawn Rotellini: Importance of Patient Advocacy at Annual Meetings https://www.mdmag.com/conference-coverage/nord-2019/importance-patient-advocacy-annual-meetings Thu, 24 Oct 2019 15:06:00 UTC Eculizumab for Neuromyelitis Optica Spectrum Disorder https://www.mdmag.com/conference-coverage/nord-2019/eculizumab-neuromyelitis-optica-spectrum-disorder Thu, 24 Oct 2019 14:13:00 UTC Study Finds Rare Diseases Still Underrepresented in Medical Journals https://www.mdmag.com/conference-coverage/nord-2019/study-rare-diseases-underrepresented-medical-journals Thu, 24 Oct 2019 12:56:00 UTC Moira Gunn, PhD: Role of Primary Care in Rare Disease https://www.mdmag.com/conference-coverage/nord-2019/primary-care-rare-disease Wed, 23 Oct 2019 21:33:00 UTC Dawn Rotellini: Changing Disease State of Hemophilia https://www.mdmag.com/conference-coverage/nord-2019/changing-disease-state-hemophilia Wed, 23 Oct 2019 17:16:00 UTC Lucas Kempf, MD: FDA and Rare Disease Therapies https://www.mdmag.com/conference-coverage/nord-2019/fda-rare-disease-therapies Wed, 23 Oct 2019 15:59:00 UTC FDA Clears Phase I PKD Study https://www.mdmag.com/medical-news/fda-pkd-study Tue, 22 Oct 2019 14:42:00 UTC FDA Approves Triple Therapy to Treat Cystic Fibrosis https://www.mdmag.com/medical-news/fda-triple-therapy-cystic-fibrosis Mon, 21 Oct 2019 19:33:00 UTC FDA Approves Romiplostim sBLA for ITP https://www.mdmag.com/medical-news/fda-romiplostim-sbla-itp Sat, 19 Oct 2019 17:47:00 UTC Minocycline Topical Foam Approved for Acne Vulgarism in Patients 9 and Older https://www.mdmag.com/medical-news/amzeeq-minocycline-topical-foam-approved-acne-vulgarism Fri, 18 Oct 2019 21:30:00 UTC FDA Approves Efficacy Trial for Novel Glioblastoma Drug https://www.mdmag.com/medical-news/fda-trial-glioblastoma-drug Tue, 15 Oct 2019 22:11:00 UTC FDA Approves Afamelanotide to Treat Erythropoietic Protoporphyria https://www.mdmag.com/medical-news/fda-afamelanotide-erythropoietic-protoporphyria Tue, 08 Oct 2019 17:52:00 UTC Gail Rodich, MD: Rituximab Approval and Its Impact https://www.mdmag.com/conference-coverage/ccr-west-2019/rituximab-approval-impact Sat, 28 Sep 2019 17:33:00 UTC FDA Approves Rituximab for Pediatric GPA, MPA https://www.mdmag.com/medical-news/fda-rituximab-pediatric-gpa-mpa Fri, 27 Sep 2019 18:41:00 UTC FDA Approves Daratumumab for ASCT-Eligible Multiple Myeloma https://www.mdmag.com/medical-news/fda-daratumumab-asct-multiple-myeloma Thu, 26 Sep 2019 18:22:00 UTC FDA Approves Nintedanib for SSc-ILD https://www.mdmag.com/medical-news/fda-nintedanib-ssc-ild Fri, 06 Sep 2019 19:43:00 UTC FDA Approves Fedratinib for Forms of Myelofibrosis https://www.mdmag.com/medical-news/fda-approves-fedratinib-for-forms-of-myelofibrosis Fri, 16 Aug 2019 14:52:00 UTC Evinacumab Cuts LDL Cholesterol in Phase 3 HoFH Trial https://www.mdmag.com/medical-news/evinacumab-ldl-cholesterol-phase-3-hofh Wed, 14 Aug 2019 15:09:00 UTC FDA Reviewing Manipulated Data from Zolgensma SMA Application https://www.mdmag.com/medical-news/fda-reviewing-manipulated-data-from-zolgensma-sma-application Tue, 06 Aug 2019 21:26:00 UTC NDA Submitted for Elagolix for Treatment of HMB Associated with Uterine Fibroids https://www.mdmag.com/medical-news/nda-elagolix-treatment-uterine-fibroids Mon, 05 Aug 2019 16:44:00 UTC FDA Approves Pexidartinib as First TGCT Therapy https://www.mdmag.com/medical-news/fda-pexidartinib-first-tgct-therapy Fri, 02 Aug 2019 20:06:00 UTC Aspirin Helps Improve Liver Function for Hepatocellular Carcinoma Patients https://www.mdmag.com/medical-news/aspirin-liver-function-hepatocellular-carcinoma Fri, 02 Aug 2019 16:16:00 UTC