Rare diseases Press Release

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01/05/2018 - 05:30
Researchers shed light on how Alzheimers processes spread through the brain
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Alzheimers Research
01/05/2018 - 00:19
Advances in brain imaging settle debate over spread of key protein in Alzheimers
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University of C...
01/02/2018 - 15:36
Alexion and Elliott Agree to Collaborate on Identifying a New Board Member
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Alexion
01/02/2018 - 15:32
Study reveals reversibility of Friedreichs ataxia in mice
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UCLA
12/26/2017 - 04:49
Soliris (Eculizumab) Receives Marketing Authorization in Japan for the Treatment of Patients with Generalized Myasthenia Gravis (gMG)
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Alexion
12/22/2017 - 07:49
BioMarin Receives Anticipated Notification of PDUFA Extension for Pegvaliase Biologics License Application (BLA) to May 28, 2018
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BioMarin
12/21/2017 - 13:45
Tax Reform Bill Expected to Be Signed Into Law
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Cystic fibrosis
12/19/2017 - 18:54
BioMarin Doses First Patient in Global GENEr8-1 Phase 3 Study of Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A
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BioMarin
12/18/2017 - 03:47
Sanofi and Alnylam submit Marketing Authorization Application to the European Medicines Agency for patisiran for the treatment of hereditary ATTR amyloidosis
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Genzyme
12/15/2017 - 05:34
FDA lifts clinical hold on fitusiran
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Genzyme
12/14/2017 - 07:10
Amicus Therapeutics Submits New Drug Application to U.S. FDA for Migalastat for Treatment of Fabry Disease
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amicus
12/12/2017 - 22:22
Sanofi and Regeneron announce positive topline pivotal results for PD-1 antibody cemiplimab in advanced cutaneous squamous cell carcinoma
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Genzyme
12/12/2017 - 20:49
Once-Daily, Oral LIXIANA (edoxaban) Met Primary Endpoint in Investigational Hokusai-VTE CANCER Study
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Daiichi Sankyo
12/12/2017 - 18:06
Daiichi Sankyo and Puma Biotechnology Announce Research Collaboration with Major Cancer Center in HER2-Mutated Cancer
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Daiichi Sankyo
12/11/2017 - 14:32
ALS Patients Lung-function Decline Scores Can Predict Breathing Failure, Study Reports
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Cytokinetics
12/11/2017 - 11:13
BioMarin Highlights New Results for Gene Therapy Valoctocogene Roxaparvovec at the 2017 American Society of Hemophilia (ASH) Meeting
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BioMarin
12/11/2017 - 10:29
Results of Phase 1b/2 Dose Regimen Optimization Studies for ALXN1210 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Presented at American Society of Hematology (ASH) Meeting
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Alexion
12/09/2017 - 07:24
BioMarin Provides 1.5 years of Clinical Data for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A at 59th American Society of Hematology (ASH) Annual Meeting Concurrent with NEJM Publication
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BioMarin
12/07/2017 - 18:35
Daiichi Sankyo Presents Updated Data for DS-8201 in Patients with HER2-Expressing Breast Cancer at San Antonio Breast Cancer Symposium
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Daiichi Sankyo
12/07/2017 - 05:56
Sanofi explores combination treatments for multiple myeloma in new late-stage trials
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Genzyme
12/07/2017 - 03:45
Alexion and Halozyme Enter License Agreement for ENHANZE Technology
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Halozyme
11/30/2017 - 20:39
Daiichi Sankyo and Translational Sciences Conclude Agreement, after Successful Phase 1a Trial, as Part of Daiichi Sankyo Portfolio Prioritization
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Daiichi Sankyo
11/30/2017 - 11:15
Designer molecule points to treatment for diseases caused by DNA repeats
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University of W...
11/30/2017 - 04:34
New Publication Shows Respiratory Decline Correlated With Disease Progression in People Living With ALS
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Cytokinetics
11/30/2017 - 01:31
Elias Zerhouni, President of Sanofi R&D, wins Executive of the Year at annual Scrip Awards
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Genzyme
11/27/2017 - 10:30
BioMarin Sells Second Priority Review Voucher for $125 Million
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BioMarin
11/21/2017 - 04:11
ArQule Announces Dosing in a Registrational Trial of FGFR Inhibitor, Derazantinib, for Treatment of Intrahepatic Cholangiocarcinoma
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ArQule
11/20/2017 - 22:44
Daiichi Sankyo Initiates Pivotal Phase 2 Study of DS-8201 in Patients with HER2-Positive Advanced Gastric Cancer
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Daiichi Sankyo
11/20/2017 - 11:30
Three UCLA professors named fellows of the American Association for the Advancement of Science
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UCLA
11/16/2017 - 12:40
FDA approves new treatment to prevent bleeding in certain patients with hemophilia A
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FDA
11/16/2017 - 06:23
100 days to go get involved in Rare Disease Day 2018!
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EURORDIS
11/16/2017 - 04:40
Trelegy Ellipta once-daily single inhaler triple therapy gains marketing authorisation in Europe for the treatment of COPD
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GlaxoSmithKline
11/15/2017 - 09:12
Ampio Announces Start of Open Label Extension Study to Support Commercial Label of Repeat Administration of Ampion in Severe Osteoarthritis of the Knee
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Ampio Pharmaceu...
11/15/2017 - 08:31
FDA approves treatment for rare genetic enzyme disorder
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FDA
11/14/2017 - 19:46
New LIXIANA sub-analysis from the ENGAGE AF-TIMI 48 trial presented at the American Heart Association Annual Scientific Sessions 2017
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Daiichi Sankyo
11/14/2017 - 09:28
Epidarex Capital company, Apellis Pharmaceuticals, completes Initial Public Offering
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VABIO
11/12/2017 - 21:29
Fujifilm invests in the bioventure, TOKIWA-Bio Inc.
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Fujifilm
11/09/2017 - 10:04
Lysosomal Acid Lipase Deficiency
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American Liver ...
11/09/2017 - 05:27
Sanofi, Principia agree to develop multiple sclerosis drug candidate
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Genzyme
11/08/2017 - 16:33
Alexion Appoints Francois Nader, M.D. to its Board of Directors
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Alexion
11/08/2017 - 10:33
Patrick Vallance, President, R&D, GSK to become UK Government's Chief Scientific Adviser
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GlaxoSmithKline
11/08/2017 - 07:03
ArQule Announces $9.5 Million Private Placement of Preferred Stock
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ArQule
11/06/2017 - 16:03
, the professional society for health economics and outcomes research (HEOR), held several sessions on the topic of orphan drugs at its
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ISPOR
11/06/2017 - 15:42
Orphan DrugsExploring the Challenges of Pricing, Reimbursement, and Funding
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ISPOR
11/06/2017 - 10:53
Cystic Fibrosis Foundation Awards Sen. Susan Collins the Breath of Life Legislator Award
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Cystic fibrosis
11/06/2017 - 08:11
Alexion Receives New Japanese Patent for Soliris (eculizumab), Extending Patent Protection Into 2027 and Strengthening Global Patent Portfolio
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Alexion
11/06/2017 - 05:38
European Conference on Rare Diseases & Orphan Products 2018 Vienna
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EURORDIS
11/06/2017 - 05:20
New international consortium to accelerate drug discovery in kidney diseases
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University of B...
11/03/2017 - 04:11
New Interim Data Presented at NASPGHAN 2017 Meeting Show Survival Beyond 1 Year of Age in Infants with LAL-D Treated with Kanuma (sebelipase alfa)
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Alexion
11/02/2017 - 07:40
Sanofi and Alnylam present positive complete results from APOLLO Phase 3 study of investigational patisiran in hereditary ATTR (hATTR) amyloidosis patients with polyneuropathy
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Genzyme
11/02/2017 - 06:45
Boehringer Ingelheim and Dicerna Announce Collaboration to Develop Novel Treatments for Chronic Liver Diseases including Nonalcoholic Steatohepatitis (NASH)
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Boehringer-Ingelheim
11/01/2017 - 08:25
New Phase 1/2 Study Data for ALXN1210 as a Potential Future Treatment for Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) to Be Presented at American Society of Hematology (ASH) Meeting
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Alexion
11/01/2017 - 06:31
FDA Grants Rare Pediatric Disease Designation to ArQule's Miransertib (ARQ 092) for the Treatment of Proteus Syndrome
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ArQule
10/31/2017 - 04:10
Dupilumab Significantly Reduced Steroid Use, Asthma Attacks, and Improved Lung Function in a Phase 3 Study of People with Severe Steroid-Dependent Asthma
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Genzyme
10/29/2017 - 23:18
Daiichi Sankyo and Glycotope Announce Option Agreement for Antibody Drug Conjugate Strategic Collaboration and Licensing
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Daiichi Sankyo
10/27/2017 - 04:58
Treatment Effects Maintained Over Five Years in Patients with Relapsing Remitting Multiple Sclerosis who Received Sanofi Genzymes Lemtrada (alemtuzumab) in Extension Study After Switching from Interferon Beta-1a
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Genzyme
10/26/2017 - 06:49
New Data Suggest Slowing of Cortical Gray Matter Atrophy with Sanofi Genzymes Aubagio (teriflunomide) is Associated with Delayed Conversion to Clinically Definite Multiple Sclerosis
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Genzyme
10/26/2017 - 06:07
Questions and Answers on 10 years of the EU Paediatric Regulation
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European Commission
10/26/2017 - 05:41
State of children's medicines in the EU
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European Commission
10/25/2017 - 07:59
Regeneron Announces 300 Additional Jobs in Limerick
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Idaireland