Articles

Rare Advocate
February 21, 2019
Rare Leader: Meghan Yarnall, Founder, Show Me Your Genes
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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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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

February 21, 2019
Enzyvant Names Alexion Executive as CEO
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February 4, 2019
MeiraGTx Enters Gene Therapy Collaboration with Ja...
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February 1, 2019
PTC Therapeutics Launches $500,000 Funding Program...
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January 31, 2019
FDA Grants RegenxBio Rare Pediatric Disease Design...
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January 31, 2019
Rare Leader: Nadia Bodkin, Trailblazer of Rare Adv...
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January 30, 2019
Study Suggest Imaging Technology Could Measure Dis...
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