Source MedCity News Interim Phase I/II data for Zolgensma showed improvements in children aged 2 to less than 5 with spinal muscular atrophy. The therapy received FDA approval in May for infants younger than 2. A gene therapy approved for treating spinal muscular atrophy in infants has also shown improved efficacy in older children. Basel,
Source University of Miami Health System Neuroscientists have been struggling for years to understand why neurons in the brain and spinal cord have so much difficulty re-growing connections after injury caused by trauma or disease. An intercontinental collaboration between the University of Miami and Imperial College London neuroscientists and computer scientists provides new insight into
The peptide nucleic acid-based technique offers an alternative to CRISPR-Cas9 A research team from Carnegie Mellon University and Yale University will advance their innovative, synthetic nucleic acid-based gene editing technique under a new grant from the National Institutes of Health’s (NIH’s) Somatic Cell Genome Editing (SCGE) Program. “Our PNA technique offers a promising avenue for treating — and possibly curing —
Terumo BCT’s latest cell therapy technology, the Finia® Fill and Finish System, is a first-of-its-kind device developed to help bring reproducibility and control to cell therapy manufacturing to get therapies to more patients who need them. Finia is a fully automated, modular, functionally closed system that creates the final formulation of cell and gene therapies
Penn study shows delivering a “distant cousin” of a key protein prevented muscle damage without triggering an immune response in large animal models A gene therapy being developed at Penn Medicine to treat Duchenne muscular dystrophy (DMD) successfully and safely stopped the severe muscle deterioration associated with the rare, genetic disease in both small and large animal
Twenty million Americans suffer from peripheral nerve injuries, which can be caused by traumas such as combat wounds and motorcycle crashes as well as medical disorders including diabetes. These injuries can have a devastating impact on quality of life, resulting in loss of sensation, motor function and long-lasting nerve pain. The body is capable of
Two new studies by an international team of researchers report progress in using stem cells to develop new therapies for Pelizaeus-Merzbacher disease (PMD), a rare genetic condition affecting boys that can be fatal before 10 years of age. Often diagnosed at birth with symptoms of weakness and breathing difficulties, the progressive neurological problems in PMD
Of an estimated 6,500 to 7,000 known rare diseases, only a fraction – maybe 5% – have U.S. Food and Drug Administration-approved treatments. To increase that percentage, the National Institutes of Health has awarded approximately $31 million in grants in fiscal year 2019 to 20 teams – including five new groups — of scientists, clinicians,
Interview with Dr. John Kennedy chief of the division of foot and ankle surgery and professor in the department of orthopedic surgery at NYU Langone in New York City. Here, Dr. Kennedy shares his insight into the future of platelet-rich plasma and stem cell therapy in sports medicine. Question: How do you see platelet-rich plasma
The number of cancer cell therapies has increased considerably over the last few years.1 There are several different types of cellular therapies, including chimeric antigen receptor (CAR) T cells, multiple tumor-associated antigen-specific T cells (TAA-T), natural killer (NK)-based therapies and T-cell therapies based on novel technologies like CRISPR.1 Amongst the different types of cell therapies in development,
