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MDA Summer Camp’s New Adventure: Going Virtual
In March, as the novel coronavirus spread across the US, we at MDA realized it would be impossible to safely gather our community’s children and teens at MDA Summer Camps…
Tags: COVID-19, Summer Camp
A Letter from Case Worker Jana Unislawski to the MDA Community: How to Create an Emergency Care Plan for the COVID-19 Era
MDA Care Center providers across the country are working on the frontlines of the novel coronavirus pandemic. Every day, they’re fielding patient questions and finding new ways to practice medicine…
Tags: COVID-19, Caregiving, MDA Care Centers
Hacking COVID: Voices from the Community, Part 2
In April, MDA fielded a survey to ask its community how COVID-19 was impacting their lives. We heard your responses — anxiety, questions, hope — and wanted to know more.…
Tags: COVID-19, Mental Health, Relationships
NS Pharma Announces Positive Results from Phase 2 Study of Viltolarsen in DMD
On May 26, NS Pharma, a wholly owned subsidiary of the Japanese company Nippon Shinyaku Co. Ltd., announced positive results from its phase 2 clinical trial of the investigational therapy…
Hacking COVID: Voices from the Community, Part 1
In April, MDA fielded a survey to ask its community how COVID-19 was impacting their lives. We heard your responses — anxiety, questions, hope — and wanted to know more.…
Tags: COVID-19, Mental Health, Relationships
Pfizer Announces Positive Results from Phase 1b Study of PF-06939926 for Treatment of DMD
On May 15, Pfizer announced positive results from a phase 1b trial assessing treatment with the company’s investigational gene therapy PF-06939926 in ambulatory boys with Duchenne muscular dystrophy (DMD). Preliminary…
Orphazyme Announces FDA Fast Track Designation for Development of Arimoclomol for Treatment of ALS
On May 22, Danish company Orphazyme announced Fast Track designation from the US Food and Drug Administration (FDA) for development of the investigational drug arimoclomol for the treatment of amyotrophic…
Game On
MDA Let’s Play takes family fun online
Tags: Featured Content
Pompe Disease Treatment Is Moving Forward
A Q&A with Priya Sunil Kishnani, MD, MBBS
MDA’s 2020 Clinical Scientific Conference Trial News and Updates
Highlights from the virtual meeting with researchers and clinicians
Tags: Clinical Trials, Drug Development, Gene Therapy, MDA Clinical and Scientific Conference, Research