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...
Duchenne Muscular Dystrophy Treatment Access: A Global Failure of Equity, Policy, and Accountability
Duchenne muscular dystrophy (DMD) is no longer a condition defined solely by its biology. Today, it is equally defined by who can access treatment—and...
Potential Upcoming New Gene Therapies for Duchenne Muscular Dystrophy
The landscape of potential new gene therapies for Duchenne muscular dystrophy (DMD) is rapidly evolving, offering renewed hope for patients and families affected by...