Articles

Business
May 24, 2019
FDA Approves Gene Therapy Zolgensma for SMA; Novartis Sets Price at $2.1 Million
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Diagnosis
May 24, 2019
Blood Test Can Provide Earlier Diagnosis of Autism Spectrum Disorder
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Rare Advocate
May 23, 2019
Rare Leader: Rob Long, Executive Director, Uplifting Athletes
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In Rare Form
May 22, 2019
Lessons for Rare Disease Advocates from Game of Thrones
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Deals
May 21, 2019
Amryt Adds to Rare Disease Pipeline with $311 Million Acquisition of Aegerion
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Business
May 21, 2019
Reneo Raises $50 Million to Develop Therapies for Mitochondrial Diseases
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Diagnosis
May 21, 2019
Smartphone App Can Help Diagnose Huntington’s Disease
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Business
May 20, 2019
Axovant Doses First Patient in Gene Therapy Trial for GM1 Gangliosidosis
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Business
May 20, 2019
FDA Expands Use of Gattex to Include Short Bowel Syndrome
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Drug Development
May 17, 2019
Understanding the Role of Natural History Studies in Clinical Trials
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People & Organizations
May 16, 2019
Rare Leader: Karen Utley, President and Co-Founder, IFCR
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Rare Advocate
May 16, 2019
Bruce Bloom to Step Down as CEO of Cures within Reach
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In Rare Form
Business
May 15, 2019
Vertex and Kymera Enter Targeted Protein Degradation Collaboration
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Business
May 14, 2019
Solid Biosciences Reports Serious Adverse Event in DMD Trial
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Business
May 14, 2019
Celgene Receives Breakthrough Therapy Designation Kaposi Sarcoma Drug
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Business
May 14, 2019
UniQure Reports Promising Interim Data from Study of Hemophilia B Gene Therapy
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Research
May 9, 2019
A Roadmap for Rare Disease Groups
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Rare Advocate
May 9, 2019
Rare Leader Profile: Mark Dant, Executive Director of The Ryan Foundation
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Policy
May 9, 2019
Minnesota Senate Passes Legislation to Create Rare Disease Advisory Council
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Business
May 8, 2019
Pfizer Pays $340 Million for Achondroplasia Therapy Developer Therachon
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Business
May 8, 2019
Vielo Bio Reports Positive Pivotal Results of Treatment for Rare CNS Disorder
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Business
May 8, 2019
Gilead Enters Strategic Collaboration with Goldfinch Bio on Kidney Diseases
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Drug Development
May 7, 2019
FDA Approves First LEMS Drug for Children and Adds New Wrinkle to Pricing Controversy
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Drug Development
May 7, 2019
GW Pharma Reports Positive Results for CBD Drug in Patients with Rare Severe Epilepsy
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Drug Development
May 7, 2019
EC Approves Akcea and Ionis’ Waylivra for Rare Lipid Disorder
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Business
May 7, 2019
European Commission Approves BioMarin’s PKU Drug Palynziq
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Rare Advocate
April 25, 2019
Rare Leader: Amanda Moore, CEO, Angelman Syndrome Foundation
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Drug Development
April 25, 2019
Global Sales of Orphan Drug Expected to Grow at Twice the Rate of Non-Orphan Drugs
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Business
April 24, 2019
Fulcrum Sees Promise in Failed Heart Drug as Treatment for FSHD
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Legislation
April 24, 2019
ACE Kids Act to Improve Coordination of Care for Medically Complex Kids
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Diagnosis
April 23, 2019
A Case for Whole Genome Sequencing
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Drug Development
April 23, 2019
Report Predicts Sharp Jump in Clinical Development Productivity for Rare Disease Therapies
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Drug Development
April 22, 2019
Novartis Investigating Patient Death in SMA Gene Therapy Trial
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Research
April 22, 2019
Study Points to Lithium as Potential Treatment for Limb Girdle Muscular Dystrophy
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Awareness
April 19, 2019
A Young Rare Disease Advocate Discusses Life with an Undiagnosed Condition
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Rare Advocate
April 18, 2019
Rare Leader: Emily Milligan, Executive Director, Barth Syndrome Foundation
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Drug Development
April 18, 2019
St. Jude Gene Therapy “Cures” Babies with “Bubble Boy” Disease
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Uncategorized
April 17, 2019
When a Cold Is a Serious Threat
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Business
April 17, 2019
FDA Grants Ultragenyx Rare Pediatric Disease Designation for Experimental Therapy
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Business
April 16, 2019
Fibrocell Teams with Castle Creek on RDEB Gene Therapy
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Business
April 16, 2019
NICE Gives Akcea Nod for Tegsedi to Treat Ultra-Rare hAATR
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Business
April 9, 2019
FDA Notifies Zogenix of Inadequacies in Dravet Syndrome Drug Application
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Business
April 9, 2019
Cyclerion Taps Shire’s Andreas Busch as Chief Innovation Officer
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Business
April 8, 2019
Alnylam and Regeneron Enter Blockbuster Collaboration
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Business
April 8, 2019
Audentes Expands Platform and Pipeline with Programs for DMD and DM
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Business
April 5, 2019
How a Patient Group Helped Drive Drug Development in Rett Syndrome
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Uncategorized
April 4, 2019
Rare Leader: Julie Raskin, Executive Director, Congenital Hyperinsulism International
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Business
April 4, 2019
ICER Calls Spinraza Too Pricey, Urges Fair Pricing of Zolgensma
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Business
April 4, 2019
FDA Grants Abeona Fast Track Designation for Sanfilippo Type B Gene Therapy
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Uncategorized
Business
April 3, 2019
Dyne Therapeutics Raises $50 Million to Develop Therapies for Muscle Diseases
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Business
April 3, 2019
EspeRare Partners with Dermelix to Develop Treatment for XLHED
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Drug Development
April 2, 2019
Sangamo Provides Positive Updates on Hemophilia A and Beta Thalassemia Gene Therapy Trials
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Business
April 2, 2019
AveXis Expands Gene Therapy Manufacturing Capacity with Acquisition
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Business
April 1, 2019
EMA Grants Krystal Biotech PRIME Designation for DEB Gene Therapy
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Business
April 1, 2019
FDA Grants AstraZenca and Merk’s NF1 Drug Breakthrough Therapy Designation
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Drug Development
April 1, 2019
FDA Orphan Drug Designations Q1 2019
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Rare Advocate
March 29, 2019
How Misperceptions Can Create Barriers to Care
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Uncategorized
March 28, 2019
Rare Leader: Mary Anne Meskis, Executive Director, Dravet Syndrome Foundation
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Business
March 28, 2019
Polyneuron Raises $22.6 Million to Advance Autoimmune Disease Therapies
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Drug Development
March 28, 2019
Orchard Therapeutics Reports MLD Gene Therapy Shows Sustained Benefit
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Awareness
March 28, 2019
Seeing Sickle Cell as a Blood Disease
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Finance
March 27, 2019
Imara Raises $63 Million, Brings in Pfizer Rare Disease Head as CMO
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Deals
March 27, 2019
ProQR and EB Research Form Drug Company
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Research
March 27, 2019
NIH Gives $3.2 Million Grant to OMRF to Study Sarcoidosis
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Drug Development
March 26, 2019
FDA Grants Promedior Breakthrough Therapy Designation for Idiopathic Pulmonary Fibrosis Drug
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Drug Development
March 25, 2019
FDA Issues Draft Guidance on Natural History Studies for Rare Diseases
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Business
March 22, 2019
Targeting Repeat Expansion Disorders with Next-Gen Antisense Drugs
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Uncategorized
March 21, 2019
Rare Leader: Melissa Bryce Gamble, Executive Director, The Global Foundation for Peroxisomal Disorders
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Business
March 21, 2019
FDA Approves Jazz Pharmaceuticals’ Narcolepsy Drug
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Policy
March 21, 2019
Medicare Part D Spending on Specialty Drugs Climbs to $32.8B in 2015
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Business
March 21, 2019
FDA Grants Enzyvant Fast Track and Rare Pediatric Disease Designations for Farber Disease Therapy
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Business
Rare Advocate
March 20, 2019
Cure SMA and Parent Project Muscular Dystrophy Form Collaboration to Collect RWD
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Business
March 20, 2019
Alexion Teams Up with Zealand, Affibody, in Pair of Development Deals
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Business
March 20, 2019
Pfizer Takes Stake in Gene Therapy Developer Vivet
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Business
March 20, 2019
GW Pharmaceuticals Sells Priority Review Voucher for $105 Million
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Drug Development
March 18, 2019
Gottlieb Calls on Industry to Modernize Approach to Clinical Trials
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Drug Development
March 15, 2019
Getting Misfolding Proteins to Shape Up
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Rare Advocate
March 14, 2019
Rare Leader: Ashley Valentine, Co-Founder, Sick Cells
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Rare Disease
March 13, 2019
A High School Student Unlocks A Rare Disease Mystery
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Research
March 13, 2019
NDF Awards $700,000 in Grants for GNE Myopathy Research and Programs
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Policy
March 12, 2019
Report Calls on NICE to Use Greater Flexibility in Reviewing Orphan Drugs
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Business
March 8, 2019
A Different Approach to Inhibiting the Complement System
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Rare Advocate
March 7, 2019
Rare Leader: Haley Oyler, President, SETBP1 Society
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Policy
March 7, 2019
Patient Group Calls on BIO to Disavow “Price Gouging” by LEMS Drugmaker
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Business
March 6, 2019
Alnylam Reports Positive Results from Late-Stage Study of Givosiran in AHP
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Awareness
March 6, 2019
FDA to Hold Public Meeting on the Impact of Rare Diseases
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Awareness
March 6, 2019
How Maria Voermans Found Her Calling
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Drug Development
March 4, 2019
Oxford, Harrington Discovery Institute Form Center to Advance Rare Disease Therapies
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Business
March 4, 2019
Biogen to Acquire Nightstar Therapeutics for $800 Million
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Business
March 2, 2019
Perlara to Wind Down Operations
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Awareness
March 1, 2019
Looking Back in Time to Find Rare Disease Patients Today
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Awareness
February 27, 2019
A Rare Disease Gets Rock Star Treatment
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Business
February 27, 2019
Amicus Establishes Global Research and Gene Therapy Center of Excellence
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Deals
February 25, 2019
Roche Agrees to Acquire Spark Therapeutics for $4.8 Billion
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Business
February 25, 2019
Ipsen to Acquire Clementia Pharmaceuticals for up to $1.3 Billion
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Business
February 22, 2019
Ionis CEO Stanley Crooke Discusses Success as a Platform Technology Company
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Policy
February 21, 2019
Catalyst Pharmaceuticals Defends Price of LEMS Drug in Letter to Senator Bernie Sanders
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Awareness
February 21, 2019
Rare Leader: Connie Lee, President and CEO, Angioma Alliance
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Business
February 21, 2019
Avexis Plans $60 Million Expansion of Manufacturing Facility in North Carolina
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Awareness
February 21, 2019
Teaching Doctors When to See If a Horse Has Stripes
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Diagnosis
February 20, 2019
Global Commission Releases Plan for Shortening Rare Disease Diagnostic Odyssey
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Business
February 19, 2019
Nestle Health Sciences Exercises Option for Codexis PKU Treatment
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Business
February 15, 2019
Passage Bio Raises $115.5 Million to Develop Gene Therapies for CNS Disease
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Business
February 15, 2019
Orchard Looks toward Harvesting Expanded Gene Therapy Pipeline
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People & Organizations
February 15, 2019
Rare Leader: Lori Sames, CEO, Hannah’s Hope Fund
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Drug Development
February 13, 2019
Study Questions the Value of Priority Review Vouchers as Incentives
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Business
February 12, 2019
Neurogene Raises $68.5 to Advance Gene Therapies for Rare Neurological Diseases
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Genomics
February 12, 2019
Combining Proteomics with Genomics Helps Pinpoint a Rare Disease
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Innovation
February 12, 2019
Rare Disease Groups Launch Newborn Screening Study in Australia
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Business
February 12, 2019
FDA Grants Clementia Rare Pediatric Disease Designation by FOP Drug
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Awareness
February 8, 2019
Connecting Rare Disease Patients in India and the United States
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Drug Development
February 8, 2019
Solid Biosciences Reports Disappointing Preliminary Results of DMD Gene Therapy Study
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Drug Development
February 8, 2019
First Results of In Vivo Gene Editing Trial of MPS II Treatment Show Only Small Benefit
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Calendar
February 8, 2019
Upcoming Events on the Rare Calendar
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Business
February 7, 2019
FDA Approves Sanofi’s Cablivi for Rare Blood-Clotting Disorder aTTP, the First of a New Class of...
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Rare Advocate
February 7, 2019
Rare Leader: Liz Marfia-Ash, President and Founder, GRIN2B Foundation
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Awareness
Policy
February 5, 2019
Sen. Bernie Sanders Investigates $375,000 Price of LEMS Drug
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Business
February 4, 2019
Alexion and Caelum Enter $550M Collaboration in AL Amyloidosis
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Rare Disease
February 4, 2019
UK Grant Supports Diagnosis and Treatment of Rare Diseases
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Innovation
February 4, 2019
ASCO Names Progress in Treating Rare Cancers Advance of the Year
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Business
February 1, 2019
Regenerative Medicine Moves into the Spotlight
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News

May 16, 2019
Peroxisomal Disorders Reach New Heights with Help ...
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May 10, 2019
How Two Mothers Discovered That a Drug for One Rar...
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May 6, 2019
AveXis’ Gene Therapy Shows Promise Across Spectr...
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May 6, 2019
Pfizer Wins FDA Approval for Rare Heart Disease D...
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May 3, 2019
Shortening the Diagnostic Odyssey
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May 2, 2019
Rare Leader: Caryl Harris, Executive Director, Ave...
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