October 10, 2019
Alexion Pays Stealth $30 Million for Option to Co-Develop Late-Stage Program in Mitochondrial Diseases
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October 8, 2019
FDA Approves First Treatment to Increase Pain-Free Light Exposure in Rare Disorder
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October 8, 2019
Avrobio Doses First Patient in Gene Therapy Trial for Cystinosis
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October 4, 2019
Sarepta Therapeutics Reports Positive Results for Limb-Girdle Gene Therapy
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October 4, 2019
Nippon Shinyaku Files for Approval of DMD Exon-Skipping Drug
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October 2, 2019
Biogen Reports Positive Results for Pre-symptomatic Treatment with Spinraza in SMA
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October 2, 2019
Two Drug Developers Targeting Rare Muscle Diseases Name New CEOs
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September 30, 2019
FDA Approves Ipsen’s Dysport to Treat Upper Limb Spasticity in Children
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September 27, 2019
Why a Big Pharma Exec Embraced Rare Neurological Diseases
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September 22, 2019
Bluebird Bio Reports Positive Long-Term Data from CALD Gene Therapy Study
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September 19, 2019
Strongbridge Biopharma Reports Late-Stage Results for Experimental Cushing’s Therapy
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September 18, 2019
Versantis Raises $16.1 Million to Develop Treatment for Liver Disease Including ACLF
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