Weekly Spotlight - 27.11.24

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Latest News

Early Myocardial Changes in DMD Patients

Early fibrofatty replacement in Duchenne muscular dystrophy (DMD) patients suggests a need for earlier cardiac interventions. The study found myocardial changes in patients with normal heart function, indicating potential for earlier treatment. Future research should focus on consistent tissue sampling to better understand cardiac progression in DMD. 

Gene Therapy Advances for Muscular Dystrophy Patients

Melissa Spencer, PhD, highlights the need for improved gene therapy for Duchenne muscular dystrophy. She emphasises the importance of standardised assays to screen for immune responses to AAV exposure. Future advancements should focus on better AAV vectors and preclinical models to enhance treatment efficacy and patient outcomes.

Novartis invests in gene therapy for muscular dystrophy treatment

Novartis has acquired Kate Therapeutics for $1.1 billion, focusing on gene therapy for muscular dystrophies like DMD, FSHD, and DM1. This move aims to learn from past industry challenges and enhance Novartis' neuromuscularcapabilities, building on their experience with therapies like Zolgensma. 

Viltolarsen Offers Hope for Duchenne Muscular Dystrophy Patients Medical Research 

Viltolarsen, a gene therapy for Duchenne muscular dystrophy, shows promise in slowing disease progression and maintaining motor function. A study found that consistent weekly treatment led to better outcomes in patients, despite some mild side effects. Further research is needed to confirm long-term efficacy and safety.