Articles

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

March 14, 2019
Strongbridge Biopharma Positive Results from Pivot...
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March 1, 2019
Sarepta Reports Early Positive Results from LGMD G...
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February 27, 2019
FDA Grants Breakthrough Therapy Designation to Ami...
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February 25, 2019
Congenital Hyperinsulinism International to Award ...
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February 21, 2019
Rare Leader: Meghan Yarnall, Founder, Show Me Your...
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February 21, 2019
Enzyvant Names Alexion Executive as CEO
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