Rare diseases Press Release

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07/16/2018 - 16:55
Baylor Chapter back to tackling rare diseases, announces Lift For Life event on July 25th
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Uplifting Athletes
07/12/2018 - 23:24
Notre Dame Chapter announces second annual Lift For Life event on Friday
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Uplifting Athletes
07/12/2018 - 22:13
Aplastic anemia, Princeton football program and former Tigers RB Jordan Culbreath have a long-time history
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Uplifting Athletes
07/12/2018 - 20:53
FDA approves the first drug with an indication for treatment of smallpox
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FDA
07/11/2018 - 02:13
New England Journal of Medicine Publishes Final Results from Innovative Phase 2 Study Showing Vaccines Can Reduce Rate of Sustained TB Infections in Adolescents at High Risk
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Aeras
07/09/2018 - 19:30
Northwestern Chapter announces Lift For Life on Thursday
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Uplifting Athletes
07/09/2018 - 08:15
NC State Chapter announces annual Lift For Life to take place on Wednesday, July 18 inside Close-King Indoor Practice Facility
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Uplifting Athletes
07/09/2018 - 06:04
Syracuse Chapter announces annual Lift For Life on Friday, July 20 inside the Manley Field House
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Uplifting Athletes
07/03/2018 - 04:49
Penn State Chapter 2018 Lift For Life raises more than $93,000 in support of the Rare Disease Community
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Uplifting Athletes
06/28/2018 - 17:29
Family travels 7,500 miles to save sons life with treatment developed at UCLA
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UCLA
06/28/2018 - 15:41
Provincial/Territorial Health Ministers Meeting Communique
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SDCL
06/27/2018 - 23:26
Alexion Submits Application for Approval of ALXN1210 as a Treatment for Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) in the European Union (EU)
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Alexion
06/27/2018 - 19:44
BioMarin Announces First Recipients of RARE Scholars Scholarship Program
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BioMarin
06/26/2018 - 23:48
Progress toward improved Wilsons disease drug
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CSHL
06/25/2018 - 22:23
Illinois Chapter announces annual Lift For Life event to take place on Saturday
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Uplifting Athletes
06/24/2018 - 18:56
FDA approves first drug comprised of an active ingredient derived from marijuana to treat rare, severe forms of epilepsy
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FDA
06/19/2018 - 20:56
Global Biotechnology leader CSL Behring becomes title sponsor for inaugural Uplifting Athletes Young Investigator Draft
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Uplifting Athletes
06/19/2018 - 19:51
Scientists unravel DNA code behind rare neurologic disease
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UTSouthwestern
06/19/2018 - 18:00
Alexion Submits Application for Priority Review and Approval of ALXN1210 as a Treatment for Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) in the U.S.
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Alexion
06/18/2018 - 23:05
Women'secret, Mendelian and New Food are honored for the most innovative initiatives of the year
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PRISA
06/15/2018 - 08:14
ArQule Presents Results from Ongoing Phase 1 Dose Escalation Study of its Reversible BTK Inhibitor, ARQ 531
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ArQule
06/15/2018 - 07:39
Novartis survey uncovers real-world impact of immune thrombocytopenia or ITP, a rare blood disease, on patients' quality of life
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Novartis
06/14/2018 - 13:31
Alexion Announces Selection of Positive Phase 3 Clinical Data for ALXN1210 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) for Late-Breaking Oral Presentation at Annual Conference of the European Hematology Association (EHA)
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Alexion
06/13/2018 - 11:58
CRISPR research tackles rare genetic disorder in children
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Michigan State ...
06/13/2018 - 11:36
Four-time Super Bowl champion and former Penn State All-American gives difficult to diagnose rare disorder a platform
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Uplifting Athletes
06/10/2018 - 20:38
Alexion and Complement Pharma Announce Partnership to Co-Develop Pre-Clinical Complement Inhibitor for Neurodegenerative Disorders
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Alexion
06/10/2018 - 19:22
Penn State Chapter 16th annual Lift For Life set for June 30 at 11 a.m. inside Holuba Hall
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Uplifting Athletes
06/07/2018 - 11:53
ArQule to Present Clinical Data for its BTK Inhibitor, ARQ 531, at the 23rd Congress of the European Hematology Association (EHA)
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ArQule
06/07/2018 - 09:54
CRF Health Industry Luminaries to Share eSource Expertise in Clinical Trials at DIA 2018
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CRF Health
06/07/2018 - 08:36
FDA Approves Genentechs Rituxan (Rituximab) for Pemphigus Vulgaris
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Genentech
06/05/2018 - 01:25
UT Southwestern experts help paralyzed college student walk again
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UTSouthwestern
06/04/2018 - 13:10
New England Journal of Medicine publishes pivotal cemiplimab trials showing positive results in advanced cutaneous squamous cell carcinoma
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Genzyme
05/31/2018 - 13:03
Daiichi Sankyo Initiates Phase 2 Study of DS-8201 in Patients with Advanced HER2-Overexpressing or HER2-Mutated Non-Squamous Non-Small Cell Lung Cancer
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Daiichi Sankyo
05/30/2018 - 16:23
Amicus Therapeutics Launches Galafold (Migalastat) for Fabry Disease in Japan
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amicus
05/30/2018 - 12:24
CRISPR Therapeutics and Vertex Provide Update on FDA Review of Investigational New Drug Application for CTX001 for the Treatment of Sickle Cell Disease
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Vertex
05/30/2018 - 08:31
Blame the mothers gene: discovery for a blinding canine eye disease reveals an unprecedented mode of inheritance
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HelsinkiUniv
05/30/2018 - 02:50
MS Progression Linked to Grey Matter Atrophy
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Celgene
05/29/2018 - 19:34
FDA expedites review of Novartis drug Promacta for first-line severe aplastic anemia (SAA)
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Novartis
05/24/2018 - 23:02
Alexion Offer for Wilson Therapeutics Accepted
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Alexion
05/23/2018 - 07:24
FDA Grants Breakthrough Therapy Designation for Tafamidis for the Treatment of Patients with Transthyretin Cardiomyopathy
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Pfizer
05/23/2018 - 05:39
Inaugural Young Investigator Draft hosted by Uplifting Athletes coming August 18 at Lincoln Financial Field
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Uplifting Athletes
05/22/2018 - 15:49
ISPOR Panel Discusses Recommendations and Rationale of Making Medicines Affordable: A National Imperative
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ISPOR
05/22/2018 - 08:14
FDA to Review Zynquista (sotagliflozin) as Potential Treatment for Type 1 Diabetes
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Lexicon Pharmac...
05/21/2018 - 12:52
New England Journal of Medicine publishes two positive Phase 3 trials showing Dupixent (dupilumab) improved moderate-to-severe asthma
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Genzyme
05/21/2018 - 11:04
FDA approves new drug for patients with chronic liver disease who have low blood platelets and are undergoing a medical procedure
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FDA
05/21/2018 - 04:30
Tieto Intelligent Wellbeing is the first Nordic AI and big data platform to receive CE certification
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Tieto
05/21/2018 - 01:52
Birmingham scientists link with hospital partners in China on Data Science health project
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University of B...
05/17/2018 - 16:01
BioMarin Announces 1 Platform and 3 Poster Presentations at World Federation of Hemophilia 2018 World Congress
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BioMarin
05/16/2018 - 16:29
First positive clinical data for cemiplimab in advanced non-small cell lung cancer to be shared at ASCO
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Genzyme
05/16/2018 - 13:47
Aiming at Cancer, We Hit Bonus Targets
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Roswell Park Ca...
05/16/2018 - 08:09
Dupixent (dupilumab) showed positive Phase 3 results in adolescents with inadequately controlled moderate-to-severe atopic dermatitis
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Genzyme
05/16/2018 - 06:17
Rare Diseases: Addressing the Challenges in Diagnosis, Drug Approval, and Patient Access
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ISPOR
05/16/2018 - 05:44
Value in Health Themed Section Offers Updated Global Perspective
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ISPOR
05/15/2018 - 10:59
Enduring neuroblastoma diagnosis with daughter Leah helped Devon Still find a purpose and passion for life after football
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Uplifting Athletes
05/15/2018 - 07:38
BioMarin Announces First Patient Dosed in Phase 1/2 Study Evaluating Valoctocogene Roxaparvovec Gene Therapy in Severe Hemophilia A Patients with Pre-existing AAV5 Antibodies
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BioMarin
05/14/2018 - 20:25
Zymeworks and Daiichi Sankyo Expand Immuno-Oncology Collaboration Focused on Bispecific Antibodies
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Daiichi Sankyo
05/11/2018 - 15:41
One bridge, one phobia and one giant challenge
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QueenslandUniv
05/09/2018 - 20:34
Spinal Fluid Could be Used to Predict the Progression of Multiple Sclerosis, Study Finds
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University of B...
05/07/2018 - 13:05
Californias Stem Cell Agency Supports Orchard Therapeutics Acquisition of GSK Gene Therapy Programs for Rare Diseases
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CIRM
05/04/2018 - 09:07
FDA approves new uses for two drugs administered together for the treatment of BRAF-positive anaplastic thyroid cancer
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FDA