Muscular Dystrophy Articles & Analysis: Older
31 news found
He developed the California Center for Rare Diseases and serves as Director, and co-founded the UCLA Center for Duchenne Muscular Dystrophy and Clinical Genomics Center. He has served as PI on numerous National Institute of Health genomics projects including the Undiagnosed Diseases Network. ...
The Muscular Dystrophy Association is pleased to announce the award of an MDA Venture Philanthropy (MVP) grant totaling $233,200 to Scottsdale, ...
BynVector
Beyond CF, Vertex has a robust pipeline of investigational small molecule, cell and genetic therapies in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, pain, type 1 diabetes, alpha-1 antitrypsin deficiency and Duchenne muscular dystrophy. Founded in 1989 ...
“As Capricor further unlocks the potential of our lead asset, CAP-1002, for treating Duchenne muscular dystrophy and continues to expand its exosome platform, Xavier’s deep industry knowledge will be instrumental as we take the Company to the next stage of its evolution,” said Linda Marbán, Ph.D., CEO, Capricor. ...
Beyond CF, Vertex has a robust pipeline of investigational small molecule, cell and genetic therapies in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, pain, type 1 diabetes, alpha-1 antitrypsin deficiency and Duchenne muscular dystrophy. Founded in 1989 ...
Capricor's lead candidate, CAP-1002, is an allogeneic cardiac-derived cell therapy that is currently in clinical development for treating Duchenne muscular dystrophy. Capricor is also developing its exosome technology as a next-generation therapeutic platform. ...
Beyond CF, Vertex has a robust pipeline of investigational small molecule, cell and genetic therapies in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, pain, type 1 diabetes, alpha-1 antitrypsin deficiency and Duchenne muscular dystrophy. Founded in 1989 ...
Cell and genetic therapies are two rapidly emerging therapeutic modalities with the potential to treat the underlying cause — and even cure — several of the serious diseases Vertex is working on including sickle cell disease, beta thalassemia, type 1 diabetes and Duchenne muscular dystrophy. Planned Leiden Center Expansion (20-22 Drydock Avenue) ...
Team Gleason and Synchron, an endovascular brain computer interface company, today announced a partnership agreement to provide patient-focused feedback on their platform to transform daily life for patients with physical and speech disabilities like ALS, muscular dystrophy, and cerebral palsy. The partnership will enable Synchron to collaborate with Team ...
Executed Partnership with Nippon Shinyaku for the Commercialization and Distribution of CAP-1002 for Duchenne Muscular Dystrophy in the United States Upfront Payment of $30 Million Received in 1 st Quarter Strengthens Cash Position and Extends Cash Runway Positive Phase 2 Results Published in The Lancet Evaluating CAP-1002 in Late-Stage Duchenne ...
Capricor's lead candidate, CAP-1002, is an allogeneic cardiac-derived cell therapy that is currently in clinical development for treating Duchenne muscular dystrophy. Capricor is also developing its exosome technology as a next-generation therapeutic platform. ...
Beyond CF, Vertex has a robust pipeline of investigational small molecule, cell and genetic therapies in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, pain, type 1 diabetes, alpha-1 antitrypsin deficiency and Duchenne muscular dystrophy. Founded in 1989 ...
Beyond CF, Vertex has a robust pipeline of investigational small molecule, cell and genetic therapies in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, pain, type 1 diabetes, alpha-1 antitrypsin deficiency and Duchenne muscular dystrophy. Founded in 1989 ...
Capricor's lead candidate, CAP-1002, is an allogeneic cardiac-derived cell therapy that is currently in clinical development for treating Duchenne muscular dystrophy and the cytokine storm associated with COVID-19. Capricor is also developing its exosome technology as a next-generation therapeutic platform. ...
Capricor's lead candidate, CAP-1002, is an allogeneic cardiac-derived cell therapy that is currently in clinical development for treating Duchenne muscular dystrophy and the cytokine storm associated with COVID-19. Capricor is also developing its exosome technology as a next-generation therapeutic platform. ...
Beyond CF, Vertex has a robust pipeline of investigational small molecule, cell and genetic therapies in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, pain, type 1 diabetes, alpha-1 antitrypsin deficiency and Duchenne muscular dystrophy. Founded in 1989 ...
The paper titled, “ Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial ,” can be accessed here . ...
"In 2021, Capricor made significant progress on our late-stage cell therapy program for Duchenne muscular dystrophy (DMD) and COVID-19 as well as the continued expansion of our exosomes platform. ...
In addition, Vertex has a rapidly expanding pipeline of cell and genetic therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus. Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. ...
Before joining Modular Medical, O'Connor Vos served as the President and CEO of the Muscular Dystrophy Association ("MDA") for three years. She was instrumental in relaunching the organization's focus on innovations in science and care and successfully built a pharmaceutical partnership practice. ...