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Blog Post | Lifestyle, Personal Stories

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


Blog Post | Science + Research

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…

Tags: Clinical Trials, Drug Development, Exon Skipping, Research, Research Advances


Blog Post | Science + Research

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…

Tags: Clinical Trials, Drug Development, Research, Research Advances


Featured Article | Uncategorized

Game On

MDA Let’s Play takes family fun online

Tags: Featured Content


Featured Article | Science + Research

Pompe Disease Treatment Is Moving Forward

A Q&A with Priya Sunil Kishnani, MD, MBBS

Tags: Clinical Trials, Drug Development, Gene Therapy, Newborn Screening, Research, Spotlight


Featured Article | News, Science + Research

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


Featured Article | Health, Lifestyle

Coping Through a Crisis

Experts weigh in on the keys to resilience in a stressful world

Tags: Featured Content, Mental Health, Relationships


Featured Article | Advocacy, Personal Stories

All in the Family

The Zelaya family (clockwise from left): Bevsi (amazing mom), Jaime (advocate dad), Josiah (kind big brother), Nehemiah (curious younger brother), and Leah (compassionate middle sister) Why we advocate for families…

Tags: From Where I Sit, Genetic Testing, Leah, MDA Care Centers


Featured Article | Independence, Lifestyle

7 Ways to Make Life at Home Easier

How to master your tasks and practice self-care

Tags: Featured Content, Innovation, MDA Care Centers, Staying Active


Blog Post | Health, Independence

A Letter From Dr. Susan Apkon to the MDA Community: How Telehealth is Changing Medicine (and How to Prepare for It)

MDA Care Center physicians 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, Healthcare, Insurance, MDA Care Centers, Technology