Rare diseases Press Release

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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 - 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
10/24/2017 - 10:30
Notre Dame Bowling Event forges a rare bond between Irish player and patient
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Uplifting Athletes
10/24/2017 - 08:22
Michael J. Fox Foundation and Tau Consortium Announce Partnership to Develop New Imaging Tools for Devastating Neurodegenerative Diseases
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The Michael J. ...
10/23/2017 - 16:18
FDA Approves Soliris (Eculizumab) for the Treatment of Patients with Generalized Myasthenia Gravis (gMG)
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Alexion
10/18/2017 - 14:41
BioMarin Highlights Breadth of Innovative Development Pipeline at R&D Day on October 18th in New York
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BioMarin
10/18/2017 - 13:53
FDA approves CAR-T cell therapy to treat adults with certain types of large B-cell lymphoma
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FDA
10/18/2017 - 07:41
Popular Science Names BioMarin's Brineura (cerliponase alfa) One of the Top Health Innovations of 2017 with "Best of What's New" Award
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BioMarin
10/16/2017 - 07:45
Sanofi and Regeneron Announce Positive Phase 2 Study Results for Dupilumab in Patients With Active Moderate-to-Severe Eosinophilic Esophagitis
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Genzyme
10/16/2017 - 06:36
ArQule Announces $15.7 Million Private Placement of Common Stock
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ArQule
10/10/2017 - 16:00
Restless legs syndrome study identifies 13 new genetic risk variants
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University of C...
10/10/2017 - 04:39
New network of Parliamentary Advocates for Rare Diseases
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EURORDIS
10/09/2017 - 05:38
Trench Foot discovery paves way for new treatment
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UniversityOfOxford
10/06/2017 - 08:50
FDA awards 15 grants for clinical trials to stimulate product development for rare diseases
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FDA
10/06/2017 - 06:37
FDA awards six grants for natural history studies in rare diseases
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FDA
10/06/2017 - 06:31
Amicus Therapeutics Appoints Michael C. Diem, MD as Senior Vice President of Business and Corporate Development
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amicus
10/04/2017 - 08:01
U-M startup marks milestone in drug therapy for boys with Duchenne muscular dystrophy
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University of M...
10/04/2017 - 07:51
Amicus Therapeutics Announces Additional Positive Data in Pompe Disease Phase 1/2 Study at World Muscle Society
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amicus
10/02/2017 - 09:13
2017 Beyond The Trophy: Meet Clemson kicker Christian Groomes
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Uplifting Athletes
09/27/2017 - 14:57
Splunk Machine Learning Helps Recursion Pharmaceuticals Treat Genetic Diseases
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Splunk
09/26/2017 - 13:09
Cincinnati Childrens Partners with FDNA to Help Children with PACS1 Syndrome
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Cincinnati Childrens
09/26/2017 - 10:48
Fordham Chapter Touchdown Pledge Drive Saturday against Yale
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Uplifting Athletes
09/26/2017 - 09:01
Illinois Chapter Touchdown Pledge Drive Friday night against Big Ten opponent Nebraska
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Uplifting Athletes
09/26/2017 - 08:40
NEHI to Honor Massachusetts Governor Charlie Baker, Dr. Patrick Conway and Dr. Jessie Gaeta at Innovators in Health Awards
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NEHI
09/26/2017 - 08:40
Florida State Chapter Touchdown Pledge Drive Saturday at Wake Forest
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Uplifting Athletes
09/26/2017 - 08:31
Penn Chapter Touchdown Pledge Drive Saturday against Dartmouth at Franklin Field
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Uplifting Athletes
09/24/2017 - 19:15
Daiichi Sankyo Announces Positive Top-line Results from Phase 3 Study in Japan of Esaxerenone for Treatment of Essential Hypertension
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Daiichi Sankyo
09/24/2017 - 16:42
2017 Beyond The Trophy: Meet NC State tight end Cole Cook
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Uplifting Athletes
09/24/2017 - 03:20
Sharing Mayo Clinic: Pushing forward despite serious lung condition
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Mayo Clinic
09/21/2017 - 04:58
U.S. FDA Grants Orphan Drug Designation for ATB200/AT2221 for Pompe Disease
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amicus
09/20/2017 - 10:26
Maryland Chapter Touchdown Pledge Drive Saturday against UCF
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Uplifting Athletes
09/20/2017 - 10:09
Clemson Chapter Touchdown Pledge Drive Saturday against Boston College
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Uplifting Athletes