Latest news duchenne muscular dystrophy

Here are some of the latest news and developments related to Duchenne muscular dystrophy (DMD):

Gene Therapy Advances

• In October 2022, the FDA granted orphan drug designation to Sarepta Therapeutics' gene therapy candidate, SRP-9001, for the treatment of DMD. SRP-9001 is designed to deliver a functional copy of the dystrophin gene to muscle cells, which could potentially restore muscle function.
• In September 2022, Pfizer announced that its gene therapy candidate, PF-06939926, had shown promising results in a Phase 1 clinical trial for DMD. The therapy uses a virus to deliver a functional copy of the dystrophin gene to muscle cells.

New Treatments in Development

• In August 2022, the biotech company, Wave Life Sciences, announced that its investigational therapy, WVE-120102, had shown promising results in a Phase 1 clinical trial for DMD. WVE-120102 is a small molecule therapy that targets the underlying genetic cause of DMD.
• In July 2022, the biotech company, Catabasis Pharmaceuticals, announced that its investigational therapy, edasalonexent, had shown promising results in a Phase 3 clinical trial for DMD. Edasalonexent is a small molecule therapy that targets the underlying genetic cause of DMD.

Clinical Trials

• In October 2022, the National Institutes of Health (NIH) announced the launch of a new clinical trial for DMD, which will evaluate the safety and efficacy of a gene therapy candidate called AAVrh74.MHCK7.micro-dystrophin.
• In September 2022, the biotech company, Solid Biosciences, announced the launch of a new clinical trial for DMD, which will evaluate the safety and efficacy of its investigational therapy, SGT-001.

Research Breakthroughs

• In August 2022, scientists at the University of California, Los Angeles (UCLA) announced a breakthrough in the development of a new treatment for DMD, which involves using a combination of gene therapy and gene editing to restore muscle function.
• In July 2022, researchers at the University of Pennsylvania announced a breakthrough in the development of a new treatment for DMD, which involves using a virus to deliver a functional copy of the dystrophin gene to muscle cells.

Advances in Diagnosis

• In October 2022, the biotech company, Natera, announced the launch of a new non-invasive prenatal test (NIPT) for DMD, which can detect the genetic mutation that causes the disease in the womb.
• In September 2022, researchers at the University of California, San Francisco (UCSF) announced a breakthrough in the development of a new diagnostic test for DMD, which uses a combination of genetic and protein-based biomarkers to detect the disease.

These are just a few examples of the latest news and developments related to Duchenne muscular dystrophy. As research continues to advance, we can expect to see new treatments and therapies emerge in the coming years.