Rare diseases Press Release

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10/17/2018 - 06:38
Daiichi Sankyo Submits Application in Japan for FLT3 Inhibitor Quizartinib for Treatment of Patients with Relapsed/Refractory FLT3-ITD AML
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Daiichi Sankyo
10/16/2018 - 08:38
Lexicon Pharmaceuticals and Ipsen Biopharmaceuticals Canada Inc. Announce Health Canada Approval of Xermelo (telotristat Ethyl) for the Treatment of Refractory Carcinoid Syndrome Diarrhea in Combination with Somatostatin Analog Therapy
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Lexicon Pharmac...
10/16/2018 - 06:24
Dupixent (dupilumab) showed positive topline results in two Phase 3 trials of patients with chronic rhinosinusitis with nasal polyps
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Genzyme
10/15/2018 - 20:43
US FDA grants Lynparza Orphan Drug Designation for pancreatic cancer
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AstraZeneca
10/15/2018 - 10:22
Borys story: Living with metachromatic leukodystrophy
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EURORDIS
10/10/2018 - 18:35
Sanofi Presents 8-Year Data on Lemtrada (Alemtuzumab)
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Genzyme
10/10/2018 - 17:57
Sanofi presents new data on Aubagio (teriflunomide)
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Genzyme
10/09/2018 - 17:14
OCREVUS (Ocrelizumab) Data Show Early Initiation of Treatment Reduces Disability Progression over Five Years in Relapsing and Primary Progressive Multiple Sclerosis
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Genentech
10/08/2018 - 19:48
Reversing Paralysis: Stem cell therapy aims to repair spinal cords afflicted by rare disorder
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UTSouthwestern
10/07/2018 - 21:17
More Than 30 Data Presentations from Sanofi Genzymes Multiple Sclerosis Franchise to Be Featured at the 34th ECTRIMS Congress
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Genzyme
10/07/2018 - 20:22
Grifols Delivers Promise of Advancing Plasma-derived Medicines through Innovative Vision and Donor Support
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Grifols
10/07/2018 - 20:06
Libtayo (cemiplimab-rwlc) Data at ESMO 2018 Congress Provide New Insights in Six Tumor Types Under Investigation
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Genzyme
10/07/2018 - 19:12
UCLA researchers identify common molecular mechanism in two skeletal disorders
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UCLA
10/04/2018 - 07:49
BlackBerry Brings its EoT Vision to Healthcare to Improve Patient Outcomes
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BlackBerry
10/02/2018 - 23:33
Japan MHLW Grants Orphan Drug Designation to Axicabtagene Ciloleucel for Treatment of Certain Types of B-Cell Lymphoma
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Daiichi Sankyo
10/02/2018 - 07:55
Shire Joins World Thrombosis Day Campaign as Global Supporter
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ISTH
10/01/2018 - 13:48
A new way to manufacture small batches of biopharmaceuticals on demand
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MIT
09/28/2018 - 06:43
FDA approves Libtayo (cemiplimab-rwlc) as first and only treatment for advanced cutaneous squamous cell carcinoma
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Genzyme
09/27/2018 - 22:29
FDA approves a new antibacterial drug to treat a serious lung disease using a novel pathway to spur innovation
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FDA
09/26/2018 - 08:58
Alexion to Acquire Syntimmune
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Alexion
09/24/2018 - 19:23
Alexion Announces Successful Phase 3 PREVENT Study of Soliris (Eculizumab) in Patients with Neuromyelitis Optica Spectrum Disorder (NMOSD)
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Alexion
09/24/2018 - 03:35
Get ready for Touchdown Pledge Drive 2018 coming in October
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Uplifting Athletes
09/24/2018 - 00:09
Novartis announces five grant recipients of eXcellence in Ophthalmology Vision Award (XOVA) for nonprofit sustainable eye health initiatives
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Novartis
09/23/2018 - 22:18
FDA awards 12 grants to fund new clinical trials to advance the development of medical products for the treatment of rare diseases
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FDA
09/22/2018 - 02:04
Novartis to file for new Lucentis (ranibizumab) indication in retinopathy of prematurity (ROP), a rare disease in premature infants that often leads to blindness
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Novartis
09/22/2018 - 00:21
New analysis of Novartis Phase III brolucizumab (RTH258) data reinforces superior reduction of retinal fluid, a key marker of disease activity in nAMD
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Novartis
09/21/2018 - 09:57
Trelegy Ellipta receives positive CHMP opinion supporting expanded COPD indication in Europe
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GlaxoSmithKline
09/21/2018 - 00:24
Novartis announces positive CHMP opinion for one-time gene therapy Luxturna to treat children and adults with rare inherited retinal disease
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Novartis
09/20/2018 - 16:24
Willows strength: Parents work with UTSW to save daughter from deadly metabolic disease
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UTSouthwestern
09/19/2018 - 10:02
Icahn School of Medicine at Mount Sinai Hosts Seventh Annual SINAInnovations Festival and Third Health Hackathon
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Mount Sinai
09/18/2018 - 01:11
Decades later, man meets the UCLA doctor who saved his life
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UCLA
09/17/2018 - 11:51
Northwestern Chapter leader and long snapper Peter Snodgrass featured this week for 2018 Beyond The Trophy series
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Uplifting Athletes
09/17/2018 - 09:03
Orion Corporation and University of Helsinki join forces in developing new treatments for Mitochondrial Diseases and Progressive Myoclonus Epilepsy
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HelsinkiUniv
09/15/2018 - 11:51
Positive Phase 3 results presented for Dupixent (dupilumab) show significant improvement on multiple measures of disease severity in adolescents with moderate-to-severe atopic dermatitis
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Genzyme
09/13/2018 - 04:08
FDA approves new kind of treatment for hairy cell leukemia
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FDA
09/13/2018 - 03:47
Research in yeast leads to serendipitous finding about a central nervous system disorder
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Salk Institute
09/12/2018 - 14:57
FDA awards five grants to advance the development of pediatric medical devices
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FDA
09/12/2018 - 04:52
New approach improves detection of diseases at birth
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YaleUniversity
09/11/2018 - 19:19
Daiichi Sankyos FLT3 Inhibitor Quizartinib Receives Orphan Drug Designation from Japanese MHLW for FLT3-Mutated AML
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Daiichi Sankyo
09/10/2018 - 20:30
INNOVCare Project results demonstrate need for integrated care for rare disease patients
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EURORDIS
09/03/2018 - 21:58
Erika Augustine appointed Robert J. Joynt Professor
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RochesterUniv
09/03/2018 - 14:49
BioMarin Expands its Cork Facility to add Drug Product Filling capability
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Idaireland
09/03/2018 - 10:21
Cablivi (caplacizumab) approved in Europe for adults with acquired thrombotic thrombocytopenic purpura (aTTP)
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Genzyme
08/31/2018 - 07:30
Detroit Lions Zach Zenner working to cure rare disease, save kids lives
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Uplifting Athletes
08/24/2018 - 13:28
RTI International to serve as partner on nationwide screening and education program for Severe Combined Immunodeficiency
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RTI International
08/21/2018 - 23:51
FDA approves first drug for neurotrophic keratitis, a rare eye disease
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FDA
08/21/2018 - 16:33
Patients with rare brain diseases help scientists open new doors for gene therapy
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UTSouthwestern
08/20/2018 - 13:11
FDA Accepts Priority Review of ALXN1210 as a Treatment for Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) in the US
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Alexion
08/17/2018 - 07:42
No Deal Brexit could be catastrophic for health, says BMA
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Ekklesia
08/16/2018 - 03:55
Teva and Regeneron Announce Positive Topline Phase 3 Fasinumab Results in Patients with Chronic Pain from Osteoarthritis of the Knee or Hip
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Teva Pharmaceutical
08/15/2018 - 05:02
Syracuse Chapter President Sam Heckel using his rare disease journey with TTP to inspire others
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Uplifting Athletes
08/13/2018 - 14:49
Double discovery reveals insights behind brain degeneration
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QueenslandUniv
08/10/2018 - 00:08
FDA approves new treatment for a rare genetic disorder, Fabry disease
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FDA
08/03/2018 - 00:19
Selumetinib granted orphan designation in Europe for neurofibromatosis type 1
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AstraZeneca
08/01/2018 - 06:27
FDA Grants Breakthrough Therapy Designation to Daiichi Sankyos FLT3 Inhibitor Quizartinib for Relapsed/Refractory FLT3-ITD AML
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Daiichi Sankyo
07/30/2018 - 18:03
Daiichi Sankyo Enters Worldwide Licensing Agreement with Glycotope for Gatipotuzumab Antibody Drug Conjugate
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Daiichi Sankyo
07/29/2018 - 22:35
Scientists discover cause of aging-related disease in mice, then reverse its symptoms
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University of W...
07/27/2018 - 12:11
Fordham Chapter announces 2018 Lift For Life event will take place on July 31
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Uplifting Athletes
07/27/2018 - 10:41
Five innovators join the ranks of the Bakar Fellows
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University of B...
07/18/2018 - 17:58
Sanofi and REVOLUTION Medicines Launch Global Partnership to Develop Potential First-in-Class Targeted Treatments for Multiple Cancers
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Genzyme