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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…
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
Living Not Suffering
An MDA Ambassador learns to live life to the fullest
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.
2019 Lasting Impression Photo Contest Winners
Meet the winners of Quest’s Lasting Impression Photo Contest
Tags: Lasting Impression, Staying Active
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
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
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
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
Amylyx Pharmaceuticals Announces Positive Results from Phase 2 Study of AMX0035 for Treatment of ALS
On Dec. 17, Amylyx Pharmaceuticals and the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital announced positive results from the phase 2 (CENTAUR) trial assessing treatment…