May 26, 2020
Amid COVID 19 Crisis, Joshua Frase Foundation Leads Search Spanning Two Continents to Deliver Ventilator to Ailing Child in Ecuador
May 05, 2020
The Myositis Association Takes the Lead in Rare Disease in Virtual Community Building and Empowering the Patient through Telemedicine
Apr 02, 2020
Dynacure Announces EUR50M ($55M) Series C Financing
Dec 20, 2019
Stealth BioTherapeutics Provides Update on Phase 3 Trial of Elamipretide in Primary Mitochondrial Myopathy
Oct 03, 2019
Medicure Announces Preferred Pricing Agreement for Zypitamag(TM) with the AIDS Drug Assistance Program ("ADAP") Crisis Task Force
Sep 03, 2019
Dynacure Appoints David Garrett as Chief Financial Officer
Aug 08, 2019
Dynacure Receives Orphan Drug Designation from the US FDA for DYN101, an Antisense Medicine to Treat Rare Disease 'Centronuclear Myopathies'
Apr 30, 2019
Dynacure Announces Approval of Clinical Trial Application for DYN101, an Antisense Medicine to Treat Rare Disease 'Centronuclear Myopathies'
Apr 25, 2019
Stealth BioTherapeutics Completes Enrollment of Pivotal Study in Primary Mitochondrial Myopathy
Apr 15, 2019
Audentes Therapeutics Announces Upcoming Presentations at 22nd Annual Meeting of the American Society of Gene and Cell Therapy Including New Data from ASPIRO, the Phase 1/2 Clinical Trial of AT132 in Patients with X-Linked Myotubular Myopathy
Oct 24, 2018
Rare Disease Foundation takes on 2.5 Million challenge grant to fund Gene Therapy
Oct 05, 2018
Audentes Therapeutics to Provide Update on New Positive Interim Data from ASPIRO, the Phase 1/2 Clinical Trial of AT132 in Patients with X-linked Myotubular Myopathy, from the 23rd International Annual Congress of the World Muscle Society
Oct 05, 2018
Audentes Therapeutics Presents New Positive Interim Data from ASPIRO, the Phase 1/2 Clinical Trial of AT132 in Patients with X-linked Myotubular Myopathy, at 23rd International Annual Congress of the World Muscle Society
Sep 24, 2018
Audentes Therapeutics to Present New Data from ASPIRO, the Phase 1/2 Clinical Trial of AT132 in Patients with X-Linked Myotubular Myopathy, at the 23rd International Congress of the World Muscle Society
Sep 05, 2018
ARMGO Pharma Receives FDA Orphan Drug Designation for ARM210/S48168 for the Treatment of Ryanodine Receptor Type 1 Related Myopathies
Aug 21, 2018
Audentes Announces Regenerative Medicine Advanced Therapy (RMAT) Designation Granted by the FDA to AT132 for the Treatment of X-Linked Myotubular Myopathy
Aug 07, 2018
Audentes Therapeutics Reports Second Quarter 2018 Financial Results and Provides Update on ASPIRO, the Phase 1/2 Clinical Trial of AT132 in Patients with X-Linked Myotubular Myopathy
Jul 31, 2018
Audentes to Release Second Quarter 2018 Financial Results and Provide Update on ASPIRO, the Phase 1/2 study of AT132 in Patients with X-Linked Myotubular Myopathy
Jun 05, 2018
Audentes Announces Priority Medicines (PRIME) Designation Granted by the European Medicines Agency to AT132 for the treatment of X-Linked Myotubular Myopathy
May 16, 2018
Audentes Announces Continuing Positive Data from First Dose Cohort of ASPIRO, a Phase 1/2 Clinical Trial of AT132 in Patients With X-Linked Myotubular Myopathy