Omigapil, an investigational therapy by Santhera Pharmaceuticals, proved to be safe and well-tolerated in children and adolescents with congenital muscular dystrophy (CMD), in a Phase 1 clinical trial. Switzerland-based Santhera recently announced the successful completion of the CALLISTO trial (NCT01805024), which tested omigapil in pediatric patients with…
Gyula Acsadi is an expert in three of the world’s most expensive rare illnesses to treat: Duchenne muscular dystrophy (DMD), spinal muscular atrophy (SMA), and Pompe disease. Acsadi, head of neurology and rehabilitation at Connecticut Children’s Medical Center in Hartford, said he’s convinced that new therapies for these inherited…
American researchers have developed a new way of delivering a gene editing therapy that significantly increases the time a repaired gene can generate the protein that’s missing in a disease. This means that a gene therapy does not have to be repeated as much to help a person counteract an…
The use of tamoxifen and Evista (raloxifene) improved cardiac, respiratory, and skeletal muscle functions, and increased bone density in a mouse model of muscular dystrophy (MD), research from the Carolinas Medical Center suggests. The study, “Long-Term Treatment of Tamoxifen and Raloxifene Alleviates Dystrophic Phenotype and Enhances Muscle Functions of FKRP Dystroglycanopathy,”…
Magnetic nanotubes with a spear-like tip may enhance the precision and effectiveness of gene therapy delivery, a promising therapeutic strategy for many genetic diseases, including muscular dystrophy, according to UCLA researchers. The study, “Precision-Guided Nanospears for Targeted and High-Throughput Intracellular Gene Delivery,” was published in the journal ACS Nano.
The Parent Project Muscular Dystrophy (PPMD) is holding its 24th annual conference this year from June 27 to July 1 in Scottsdale, Arizona. Patients, families, physicians, industry partners, and other members of the community will gather at the event to discuss current issues related to Duchenne’s muscular dystrophy…
As new diagnostic tools and treatments help patients with Duchenne muscular dystrophy (DMD) live into adulthood, new challenges arise in areas like education, independence, personal relationships, health, and intimacy. To address the emerging needs of patients, caregivers and physicians of DMD patients, the Centers for Disease Control and…
AMO Pharma’s myotonic dystrophy treatment improved patients’ cognition, fatigue and ability to perform daily tasks, a Phase 2 clinical trial showed. The therapy, AMO-02 (tideglusib), also improved patients’ autism symptoms. In addition, it was safe and patients tolerated it well. Dr. Joseph Horrigan, the company’s chief medical officer, presented the findings at…
Scientists are increasingly looking at gene therapies as potential treatments for Duchenne muscular dystrophy. But there are a lot of ways to do them, and an expert in the field shed light on the subject in a webinar arranged by Parent Project Muscular Dystrophy (PPMD). Dr. Timothy Cripe discussed the…
Genetic sequencing and the speed with which it can help diagnose a child’s disease — in addition to revealing the genes that cause at least half of the 7,000 rare diseases currently known — was the focus of a discussion by three top New York geneticists. The Feb. 28 conference,…
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