Weekly Spotlight - 17.10.24

In the News

Duchenne Muscular Dystrophy: Emerging Therapies and Research Insights

The Duchenne Muscular Dystrophy Pipeline report offers comprehensive insights into over 75 companies and therapies, highlighting key drugs like Vamorolone and Givinostat. It assesses therapeutic advancements, unmet needs, and market dynamics, providing a detailed analysis of clinical stages and molecule types, aiming to revolutionise musculoskeletal care.

Exploring lncRNAs' Role in Duchenne Muscular Dystrophy Myoblasts

The study identifies 554 differentially expressed lncRNAs in DMD/mdx myoblasts, highlighting their potential as biomarkers and therapeutic targets for Duchenne Muscular Dystrophy. By analysing ceRNA networks, the research reveals lncRNAs' regulatory roles in key genes, offering insights into DMD progression and potential interventions.

Capricor Begins BLA Submission for Duchenne Cardiomyopathy Treatment Approval

Capricor Therapeutics has begun a rolling submission of its Biologics License Application for deramiocel CAP-1002, targeting Duchenne cardiomyopathy. This process, based on cardiac data from Phase 2 trials, marks a first in seeking approval for cardiac outcomes. Completion is expected by 2024's end.

Join the 2024 NYC Marathon to Support Duchenne Muscular Dystrophy Research

The 2024 TCS New York City Marathon, scheduled for 3rd November, offers a unique experience across all five boroughs. Joining the Race to End Duchenne team supports efforts against Duchenne Muscular Dystrophy. Participants must meet specific age, fee, and fundraising requirements. The team is currently full, with a waiting list available.

Oxford-Harrington Rare Disease Centre

David Cameron: Britain's Role in Advancing Rare Disease Treatments

David Cameron's article in The Times highlights Britain's potential to lead in treating rare diseases through genomics. Inspired by personal tragedy, Cameron reflects on the loss of his son Ivan to Ohtahara syndrome, a rare condition. He notes the rapid advancements in genomics since the early 2000s, when sequencing a genome was costly and time-consuming. Today, it is affordable and quick, enabling personalised medicine. Cameron's 100,000 Genomes Project marked a significant step in this field, and now, Genomics England and the NHS plan to sequence 100,000 newborns' genomes to identify rare diseases.

Cameron announces his role as chair of the Oxford-Harrington Rare Disease Centre's advisory council, a partnership aiming to develop 40 new treatments for rare diseases in the next decade. He emphasises Britain's strengths: a history of DNA research, a national health system, and a robust venture capital industry. Coupled with US expertise, this partnership is poised for breakthroughs in genomics.

The implications are vast. Genomics can help millions globally, as 80% of rare diseases have a genetic basis. Understanding one condition can unlock insights into others, including common killers like cancer and heart disease. Gene therapy, where Britain excels, offers potential to correct genetic defects, lessening or reversing conditions.