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Blog Post | Advocacy

MDA’s 2020 Advocacy Plan

It might only be a month into 2020, but MDA’s advocacy team and grassroots advocates are already making their voices heard. We expect the next 12 months to be crucial…

Tags: Accessible Air Travel, Advocacy Updates, Drug Development, Employment, Genetic Testing, Insurance, Newborn Screening


Blog Post | News, Science + Research

Antisense Therapeutics Announces Positive Results from Phase 2 Study of ATL1102 in DMD

Australian company Antisense Therapeutics announced encouraging results from its phase 2, open-label clinical trial of the company’s investigational therapy ATL1102 for treating Duchenne muscular dystrophy (DMD). The study met its…

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


Featured Article | Advocacy, Health

A Good Beginning: Newborn Screening

This public health program makes early treatment possible for babies born with neuromuscular diseases

Tags: Antisense Oligonucleotide, Exon Skipping, Featured Content, Genetic Testing, Newborn Screening, Research Advances


Featured Article | Personal Stories

Living Not Suffering

An MDA Ambassador learns to live life to the fullest

Tags: Ambassadors, From Where I Sit, Genetic Testing, Staying Active


Featured Article | Health, Science + Research

Reasons Why You Should Get Genetic Testing

Among the advantages of genetic testing, it can confirm a diagnosis and help predict how a disease will progress, giving vital information for treatment.

Tags: Featured Content, Genetic Testing, MDA Care Centers


Featured Article | Personal Stories

2019 Lasting Impression Photo Contest Winners

Meet the winners of Quest’s Lasting Impression Photo Contest

Tags: Lasting Impression, Staying Active


Featured Article | Advocacy, Health, Science + Research

Advancing Health, One Research Study at a Time

Mikey (left) is in a clinical trial for a DMD therapy. The medication could benefit his brother, Reid (right), and many other boys with DMD. MDA families can get involved…

Tags: Clinical Trials, Featured Content, MOVR, Research


Blog Post | News, Science + Research

MDA Awards More Than $1 Million in Venture Philanthropy Funding to AavantiBio to Develop Gene-Targeting Therapy for Friedreich’s Ataxia

AavantiBio, a biotechnology company developing a gene-targeting therapy for Friedreich’s ataxia (FA), was awarded MDA Venture Philanthropy (MVP) funding totaling $1,076,232 to advance AavantiBio’s phase 2 clinical trial of a…

Tags: Clinical Trials, Gene Therapy, Grants


Blog Post | News, Science + Research

FDA Approves ITF Pharma’s Tiglutik via PEG Tube for the Treatment of ALS

On Dec. 13, the US Food and Drug Administration (FDA) approved expanded labeling for ITF Pharma’s Tiglutik to include administration via percutaneous endoscopic gastrostomy (PEG) tube for the treatment of…

Tags: Drug Approval, Research Advances


Blog Post | News, Science + Research

Mallinckrodt Pharmaceuticals Releases Statement to DMD Community Announcing Discontinuation of Clinical Trials Program for DMD

Yesterday, Mallinckrodt Pharmaceuticals released a statement to the Duchenne muscular dystrophy (DMD) community regarding its decision to discontinue its DMD clinical development program. The full statement follows. After careful consideration and despite…

Tags: Clinical Trials, Research