June 11, 2017
Castle Creek Announces First Patient Enrolled in DELIVERS Study of Diacerein 1% Ointment for Epidermolysis Bullosa Simplex
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May 18, 2017
Abeona Therapeutics Announces New Updates on Sanfilippo Syndrome Clinical Trial
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May 14, 2017
Abeona Therapeutics Receives Regulatory Approval to Initiate Clinical Trial in Australia with ABO-102 Gene Therapy For Patients with MPS IIIA
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May 4, 2017
Powered by CIRM Grant, jCyte Launches New Clinical Trial for RP
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March 9, 2017
New Hope For Epidermolysis Bullosa Patients with Investigational Therapy from Castle Creek Pharmaceuticals
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February 14, 2017
Sanofi Genzyme’s Phase 2 Clinical Trial to Evaluate Oral Substrate Reduction Therapy for Gaucher Disease Type 3
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January 19, 2017
Why Health Insurers Won't Cover this $300,000-a-year Rare Disease Drug
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December 20, 2016
Introducing the FirST-4-LEMS Study
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November 21, 2016
Genentech’s New Giant Cell Arteritis Drug Shows Majority of Users Achieved Steroid-Free Disease Remission at One Year
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October 10, 2016
Abeona Therapeutics Authorized to Move Forward with Clinical Trial for Sanfilippo Syndrome Treatment
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September 1, 2016
ArmaGen Announces Initiation of Phase 2 Proof-of-Concept Clinical Trial in Brazil to Study AGT-181 for Treatment of Hurler Syndrome
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July 31, 2016
A New Drug for Acomegaly Patients? MPowered Offers Oral Alternative
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