New international consortium to promote stem cell-based therapy to treat Huntington’s disease

The Stem Cells for Huntington’s Disease ( SC4HD ) is a new international consortium created to drive advanced therapy drugs (ATMP), through stem cell transplantation, to treat Huntington’s disease. The entity, made up of twenty-eight renowned researchers from ten countries, has been officially featured in the Journal of Huntington’s Disease . The main objectives of the SC4HD consortium are to

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An international working group (IWG) consensus report redefines outcomes in immune TTP

Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a potentially fatal thrombotic microangiopathy caused by autoantibody-mediated severe deficiency of ADAMTS13. Standardized definitions of response, exacerbation, remission, and relapse were initially proposed in 2003 and modified by the International Working Group for TTP in 2017. These definitions, which have been widely used in clinical practice and research, are

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Orchard Therapeutics with research alliance partners Fondazione Telethon and Ospedale San Raffaele Milan Receives EC Approval for Libmeldy™ for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD)

First gene therapy to receive full EU marketing authorization for eligible MLD patients. One-time treatment with Libmeldy has been shown to preserve motor and cognitive function. Orchard Therapeutics, a global gene therapy leader, and its research alliance partners Fondazione Telethon and Ospedale San Raffaele, today announced that the European Commission (EC) granted full (standard) market authorization

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DNA-editing method shows promise to treat mouse model of progeria

Using a recently developed DNA base-editing technique, researchers correct accelerating aging disorder. Researchers have successfully used a DNA-editing technique to extend the lifespan of mice with the genetic variation associated with progeria,  a rare genetic disease that causes extreme premature aging in children and can significantly shorten their life expectancy. The study was published in the journal Nature, and was

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New inflammatory disorder called vacuoles, E1 enzyme, X-linked, autoinflammatory and somatic syndrome (VEXAS) discovered by NIH researchers

Researchers from the National Institutes of Health (NIH) have discovered a new inflammatory disorder called vacuoles, E1 enzyme, X-linked, autoinflammatory and somatic syndrome (VEXAS), which is caused by mutations in the UBA1 gene. VEXAS causes symptoms that included blood clots in veins, recurrent fevers, pulmonary abnormalities and vacuoles (unusual cavity-like structures) in myeloid cells. The scientists reported

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New Report from ARM Provides Overview of the Regenerative Medicine Landscape for Rare Disease

Rare Disease & Regenerative Medicine 2019 This report provides industry specific statistics compiled from cell therapy, gene therapy, tissue engineering, and other regenerative medicine companies active in developing therapies for rare disease and provides context for the potential of regenerative medicine to improve upon the current standard of care.


€7.45m EU Horizon 2020 Research Project for Robotic Production of Regenerative Stem Cell Therapies for Osteoarthritis

NUI Galway’s Regenerative Medicine Institute has launched a new €7.45 million project to develop ground-breaking and innovative scientific and engineering platforms for the production of advanced cellular therapeutics for use in the treatment of osteoarthritis and other major diseases.  Funded by the EU Horizon 2020 programme the AutoCRAT project will address the critical need to

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Mount Sinai Surgeons Perform First Surgery in New York City Using FDA-Approved Spinal Tethering Device

Mount Sinai surgeons have performed the first-ever spinal tethering surgery in New York City to correct idiopathic scoliosis—a sideways curvature in the spine—in children and adolescents. The procedure performed by Baron S. Lonner, MD, Professor of Orthopedic Surgery, and Pediatrics, Icahn School of Medicine at Mount Sinai, and Chief of Minimally Invasive Scoliosis Surgery, Mount

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NIH Awards $7.5 million to ramp up clinical trials for rare, genetic airway diseases

Formed 15 years ago, the Genetic Disorders of Mucociliary Clearance Consortium is now co-led by UNC’s Stephanie Davis, MD, to continue advancing knowledge and treatments for genetic chronic pulmonary conditions. Source UNC Health Care To continue the push toward clinical trial readiness for individuals with rare diseases, the National Center for Advancing Translational Sciences, in

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Autologous nonmyeloablative hematopoietic stem cell transplantation for neuromyelitis optica

Patients with neurological disease that causes blindness, disability go treatment free Source Northwestern University A stem cell transplant reversed a debilitating neurological disease that causes half of the patients to go blind and lose the ability to walk five years after diagnosis. Most of the patients stayed better five years after the transplant and were

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