September has been a rough month already for Novo Nordisk, which canned two more late-stage trials of its embattled IL-6 inhibitor, and Novartis, which took Phase 3 hits in both cardiovascular and muscular dystrophy indications; Ultragenyx’s big bet in Angelman syndrome failed to yield benefit in Phase 3 but Ionis’ Pharmaceuticals nabbed the first targeted approval for Alexander disease; and on the business side, biopharma investment is back in a big way.

Original title: “Novo’s canned heart disease trials, Novartis’ clinical fails, first approval for Alexander disease”

inpp5k related muscular dystrophy caused by a novel synonymous splicing variant in a chinese patient a case report

Original title: “INPP5K-related muscular dystrophy caused by a novel synonymous splicing variant in a Chinese patient: a case report.”

The RNA-based medicine is one of a handful of antibody-oligonucleotide conjugates that Novartis acquired last October when it took over neuromuscular-focused Avidity Biosciences.

Original title: “Novartis’ $12B Avidity buy pays dividends with Phase 1/2 muscular dystrophy win”

The Advanced Therapy Medicinal Products Market Trends are gaining momentum as healthcare systems increasingly adopt gene therapies cell based therapies and tissue engineered products for chronic and rare diseases According to industry estimates the sector is projected to grow from ... Get Sample PDF @ https://www.theinsightpartners.com/sample/TIPRE00012921?utm_source=OpenPR&utm_medium=10812 Rising Adoption of Cell and Gene Therapies Cell and gene therapies are emerging as transformative solutions for diseases that previously had limited treatment options. The increasing prevalence of cancer, hemophilia, muscular dystrophy, and rare genetic disorders is encouraging healthcare providers to adopt innovative therapeutic approaches. Gene therapies are particularly gaining attention for their ability to address the root causes of inherited diseases rather than only managing symptoms. CAR-T therapy has also demonstrated significant clinical success in oncology applications. Press release - The Insight Partners - Advanced Therapy Medicinal Products Market Size to Reach US$ 46.11 Billion by 2034, Growing at 14.79% CAGR During 2026-2034 - published on openPR.com Healthcare providers are increasingly integrating genomic testing, biomarker analysis, and AI-driven diagnostics into treatment planning. This trend is creating significant opportunities for manufacturers developing customized therapeutic solutions. AI-enabled drug discovery platforms are also reducing development timelines and improving clinical trial efficiency. North America currently dominates the global landscape due to strong healthcare infrastructure, high research spending, and supportive regulatory frameworks. The United States remains a major contributor, supported by extensive clinical trial activity and growing demand for innovative therapies.

Original title: “Advanced Therapy Medicinal Products Market Size to Reach US$ 46.11 Billion by 2034, Growing at 14.79% CAGR During 2026-2034”

sort lnps encapsulating cas9 mrna achieve efficient editing in skeletal muscle in a dystrophic mouse model

Original title: “SORT LNPs encapsulating Cas9 mRNA achieve efficient editing in skeletal muscle in a dystrophic mouse model.”

The agency acted after reviewing mouse data submitted in 2024, making the timing of the new pause “confusing,” one analyst wrote.

Original title: “PepGen muscular dystrophy drug gets ‘surprise’ hold from FDA”

UniQure and REGENXBIO are both dealing with FDA setbacks for their respective gene therapies, as regulatory experts question the FDA’s decision-making processes; CBER director Vinay Prasad is under probe for allegedly fostering a toxic workplace; Sarepta CEO Doug Ingram is stepping down after several years of tumult at the top of the muscular dystrophy–focused company; and Eli Lilly again tops Novo Nordisk in a weight loss trial.

Original title: “UniQure’s Delay, REGENXBIO’s Rejection Explained, Sarepta’s Ingram Steps Down, More”

Rare Disease Day is held on the last day of February to raise awareness for rare diseases and address drug development challenges. This was spurred by activists who lobbied Congress to take action against the drought of therapies for several rare diseases like Huntington’s disease, amyotrophic lateral sclerosis (ALS), Tourette syndrome, and muscular dystrophy. Thus, the National Organization for Rare Disorders (NORD) was formed to unite patient advocacy groups and raise awareness of rare diseases. Much of this is owed to regulatory and manufacturing hurdles that drug developers face. As people who have rare diseases belong to small patient populations, traditional clinical trial designs are “poorly suited” for rare diseases, according to Matthew Wood, professor of Neuroscience at the University of Oxford. According to the BioIndustry Association: “Developing any drug is a long and expensive process, but for rare diseases, the situation is even more difficult. Smaller patient populations make research more challenging, and the opportunity to get a return on investment smaller.” Wood expressed that there exists a “translational gap” when it comes to research and development (R&D) in the rare disease space. “World-class science exists, but (there is) insufficient infrastructure to move discoveries into drug development. OHC was established to help close that gap,” said Wood. Despite the incentives that accompanied the Orphan Drug Act, many argue that they are insufficient to meet the urgent unmet needs of patients while driving high costs.

Original title: “Rare Disease Day: is a regulatory overhaul what the therapeutic field needs?”

When Doug Ingram became Sarepta Therapeutics’ CEO in 2017, he didn’t have a connection to muscular dystrophy, but he has developed a fierce passion for the therapeutic area. He will step aside from his role to dedicate more time to his family.

Original title: “Sarepta CEO Doug Ingram To Step Down as Muscular Dystrophy Mission Hits Home”

New York, Monday, January 26, 2026 – In recognition of Rare Disease Day on February 28, the Muscular Dystrophy Association (MDA) is sharing powerful community stories throughout the month that reflect how connection, research, and advocacy are advancing care and opportunity for people living ... New York, Monday, January 26, 2026 – In recognition of Rare Disease Day on February 28, the Muscular Dystrophy Association (MDA) is sharing powerful community stories throughout the month that reflect how connection, research, and advocacy are advancing care and opportunity for people living with rare neuromuscular diseases. MDA is the #1 health nonprofit advancing research, care and advocacy for people living with muscular dystrophy, ALS, and related neuromuscular diseases. Rare Disease Day is observed globally on February 28 to prompt action through increased awareness, research, and community engagement. MDA encourages people diagnosed with a neuromuscular condition and their families to engage with its year-round programming—including educational webinars, support groups, advocacy initiatives, and community events—to stay informed and connected. Early on, her family was learning how to support a child with a rare, complex condition. Now at 23, Gabrielle is an adult building her future. She is pursuing graduate study in clinical mental health counseling, with the goal of one day opening her own clinic to serve underrepresented communities including people living with disabilities. Gabrielle credits MDA’s support in helping shape her career goals and her advocacy work.

Original title: “Muscular Dystrophy Association Marks Rare Disease Day by Spotlighting Community Stories Driving Progress in Research, Care, and Advocacy | Muscular Dystrophy Association”