If Elevidys Works for Duchenne Muscular Dystrophy, Why Is Sarepta’s Stock Falling? What Families Need to Know Before Choosing Gene Therapy
Few Duchenne muscular dystrophy (DMD) treatments have generated as much excitement, controversy, and debate as Elevidys (delandistrogene moxeparvovec), the gene therapy developed by Sarepta...
Elevidys Under Scrutiny: A Candid Interview with the Turkish Representative of DMDWarrioR on Cost, Effectiveness, and Ethics
Elevidys gene therapy has shown promising results in clinical trials for DMD. However, questions remain about its real-world effectiveness and its approximately $3 million...
Vamorolone (Agamree) Rejected by INESSS: When Cost Takes Priority Over Compassion in DMD Care
The recent decision by INESSS (Institut national d'excellence en santé et en services sociaux) in Canada to refuse reimbursement for Agamree (vamorolone) represents more...
PBGENE-DMD Gene Editing Therapy: Benefits, Eligibility, and FAQs
PBGENE-DMD Gene Editing Therapy is an investigational, first-in-class treatment developed by Precision BioSciences for Duchenne muscular dystrophy (DMD).Unlike traditional therapies, PBGENE-DMD uses a gene...
Why CK, AST, and ALT Data Matter More Than Videos in Duchenne Gene Therapy
In the evolving landscape of gene therapy for Duchenne muscular dystrophy (DMD), one issue is becoming increasingly clear: CK levels after Duchenne gene therapy...