Six patients with rare blood disease are doing well after gene therapy clinical trial
UCLA researchers are part of an international team that reported the use of a stem cell gene therapy to treat nine people with the rare, inherited blood disease known as X-linked chronic granulomatous disease, or X-CGD. Six of those patients are now in remission and have stopped other treatments. Before now, people with X-CGD – which causes recurrent infections, prolonged hospitalizations for treatment, and a shortened lifespan – had to rely on bone marrow donations for a chance at remission.“With this gene therapy, you can use a patient’s own stem cells instead of donor cells for a transplant,” said Dr. Donald K...
Source: UCLA Newsroom: Health Sciences - January 28, 2020 Category: Universities & Medical Training Source Type: news
Gene scissors against incurable muscular disease
(Technical University of Munich (TUM)) Duchenne type muscular dystrophy (DMD) is the most common hereditary muscular disease among children, leaving them wheelchair-bound before the age of twelve and reducing life expectancy. Researchers at Technical University of Munich (TUM), Ludwig Maximilian University of Munich (LMU) and the German Research Center for Environmental Health (Helmholtz Zentrum M ü nchen) have developed a gene therapy that may provide permanent relief for those suffering from DMD. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - January 27, 2020 Category: International Medicine & Public Health Source Type: news
Heart-function protein may help muscular dystrophy patients live longer
(Rutgers University) Rutgers-led discovery may help prevent muscular dystrophy-related heart disease, the leading cause of death in patients with Duchenne muscular dystrophy (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - January 14, 2020 Category: Biology Source Type: news
The impact of gun violence on those already dying: perspectives from a palliative care physician - Vente TM.
The consult was for a 19-year-old man with Duchenne muscular dystrophy who was in the ICU with respiratory distress and weight loss. Given his degenerative disease, establishing care with the palliative care team made perfect sense. It was, by all mean... (Source: SafetyLit)
Source: SafetyLit - January 8, 2020 Category: International Medicine & Public Health Tags: Commentary Source Type: news
Biotech firm makes $2.8B deal to license muscular dystrophy gene therapy
A gene therapy invented at Nationwide Children's Hospital just landed a licensing deal worth as much as $2.8 billion for a Boston biotech, and now the company's Columbus, Ohio office will play a big role in getting the treatment to the market.
Sarepta Therapeutics Inc. has licensed the commercial rights outside of the United States to the Swiss pharmaceutical giant Roche for $1.1 billion in cash and stock up front. Sarepta also can earn as much as $1.7 billion in milestone payments, plus royalties.… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - December 30, 2019 Category: Pharmaceuticals Authors: Carrie Ghose Source Type: news
