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A research team has developed and safely delivered a personalized gene editing therapy to treat an infant with a life-threatening, incurable genetic disease. The infant, who was diagnosed with the rare condition carbamoyl phosphate synthetase 1 (CPS1) deficiency shortly after birth, has responded positively to the treatment.
Results from a phase 1/2 clinical trial of a novel stem cell treatment for cornea injuries found 14 patients treated and tracked for 18 months had a more than 90% success rate at restoring the cornea's surface and improvements in vision.
Data presented at ASCO show that swapping in the oral drug camizestrant for an older therapy helped sustain the benefit of initial treatment, potentially opening a novel step in patient care.
Five years after helping launch Allogene Therapeutics, Pfizer is backing another developer of “off-the-shelf” cell therapies in a deal that gives the drugmaker the chance to acquire a multiple myeloma treatment.
A new gene therapytreatment for Duchenne muscular dystrophy (DMD) shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in the future, repairing those muscles.
In the rapidly advancing field of cell therapies, Dr Jason Bock has emerged as a leader, known for his innovative approach to optimising the development process. With over 25 years of experience in therapeutics, Bock has played a pivotal role in shaping the future of cell therapies, particularly through his work at CTMC.
Elevidys sales have increased since the FDA made a controversial choice to expand the therapy's use. Now, Sarepta is abandoning a successor to its drug Exondys 51, citing an “evolving" treatment landscape.
Food and Drug Administration on Friday approved two milestone gene therapies for sickle cell disease, including the first treatment ever approved that uses gene-editing technology. FRIDAY, Dec. 8, 2023 -- The U.S. Casgevy, developed by Vertex.
22, 2024 -- At-home brain stimulation therapy can safely and effectively treat severe to moderate depression, a new clinical trial shows.Rates of treatment response and depression remission were three times higher in people receiving. TUESDAY, Oct.
Food and Drug Administration has told drugmakers to add a boxed warning to a type of cancer treatment called CAR-T therapy, saying the treatment itself may sometimes cause a secondary. WEDNESDAY, Jan. 24, 2024 (Healthday News) -- The U.S.
22, 2024 -- Light therapy could be a useful treatment for the most common form of age-related macular degeneration, a new study says.The therapy, called photobiomodulation or “red light” therapy, can reduce the risk of vision loss and. TUESDAY, Oct.
25, 2024 -- A new gene therapy approved earlier this year can serve as a sustainable single-dose treatment for people with hemophilia B, newly published clinical trial data shows. THURSDAY, Sept. People with hemophilia B saw their bleeding episodes.
Scientists have developed new light-sensitive chemicals that can radically improve the treatment of aggressive cancers with minimal side effects. In mouse tests, the new therapy completely eradicated metastatic breast cancer tumors.
Still, the therapies’ modest benefit as a maintenance therapy points to how better treatments are needed for small cell lung cancer, according to one physician.
TUESDAY, June 18, 2024 -- There's more good news in the battle against sickle cell disease, with another trial finding CRISPR gene-editing therapy delivering impressive results for patients.“It’s It’s encouraging that this gene-editing treatment con.
11, 2024 -- Contrary to a warning placed on labels for CART-T cancer therapies, use of these treatments does not appear to boost the odds for a secondary cancer later, a new study shows.Researchers at Memorial Sloan Kettering. WEDNESDAY, Sept.
The opinion would allow Abecma to be prescribed after two standard therapies and is the latest sign of CAR-T’s growing use in earlier cancer treatment.
FRIDAY, May 17, 2024 -- Cutting-edge cancer treatments are essentially useless for patients barely clinging to life, a new study shows.Chemotherapies, immunotherapies, targeted therapies and hormone therapies do not improve survival rates in.
CRISPR-CAS9 is an innovative gene editing technology to precisely remove or incorporate into the gene of interest; CRISPR-CAS9 shows promising results in HIV treatment. This article outlines the current antiretroviral therapy and its adverse effects but also CRISPR/Cas technology.
Food and Drug Administration on Thursday approved the drug Elevidys, the first gene therapy for the treatment of children with Duchenne muscular dystrophy (DMD). The groundbreaking treatment will not be cheap: FRIDAY, June 23, 2023 -- The U.S.
Kyowa will pay nearly $400 million to acquire Orchard, which sells the gene therapy Libmeldy in Europe but has struggled to find paths to market for other experimental treatments.
“This experience underscored the gaps in rare disease innovation, motivating me to lead Alltrna, a company pioneering engineered tRNA therapies to address a broad range of genetic diseases in a profound way unlike any other genetic modality.”
An 11-year-old boy born deaf can hear after receiving Lilly's genetic medicine. Others, including biotechs in China and Regeneron in the U.S., are taking aim at the same drug target.
One expert views Amtagvi’s approval as a catalyst for further investment in TIL therapies, akin to how Kymriah’s 2017 clearance buoyed CAR-T treatment.
MONDAY, April 1, 2024 -- Telehealth might be a more effective way of treating alcoholism than in-person therapy sessions, a new study reports.Alcoholics who receive treatment through telehealth were more likely to engage in more therapy visits and.
25, 2024 -- A new gene therapy approved earlier this year can serve as a sustainable single-dose treatment for people with hemophilia B, newly published clinical trial data shows.People with hemophilia B saw their bleeding episodes. THURSDAY, Sept.
A landmark study has shown that severe asthma can be controlled using biologic therapies, without the addition of regular high-dose inhaled steroids which can have significant side effects.
Food and Drug Administration on Friday approved two milestone gene therapies for sickle cell disease, including the first treatment ever approved that uses gene-editing technology.Casgevy, developed by Vertex. FRIDAY, Dec. 8, 2023 -- The U.S.
Food and Drug Administration on Thursday approved the drug Elevidys, the first gene therapy for the treatment of children with Duchenne muscular dystrophy (DMD). The groundbreaking treatment will not be cheap: FRIDAY, June 23, 2023 -- The U.S.
The FDA-approved therapy, called Palforzia, was little used, leading Nestle to abandon a business it had secured in a $2.6 billion deal three years ago.
THURSDAY, July 13, 2023 -- A preclinical study offers a potential new therapy for treatment-resistant prostate cancer, offering new hope for men with the disease. The study used the chemotherapy drug cisplatin, administered orally, to disrupt the.
FRIDAY, June 14, 2024 -- Animal studies are often considered a first step in finding new drugs and treatments for human diseases, but a new review has discovered that precious few actually produce real-world therapies. Only 5% of therapies tested.
MONDAY, April 1, 2024 -- A monthly long-acting injection of buprenorphine can be an easier and more effective therapy for people struggling with opioid addiction, but treatment centers aren’t much interested in using it, a new study.
The high-profile meeting focused on the theoretical risks of CRISPR gene editing, as both the FDA and its advisory committee appeared convinced by the efficacy of the companies’ exa-cel treatment.
THURSDAY, June 27, 2024 -- Acupuncture can help breast cancer patients deal with the hot flashes that often accompany hormone therapy, a new clinical trial says.Nearly 2 in 3 women who got acupuncture during hormone therapy reported fewer and less.
14, 2023 -- For people with severe alcohol use disorder, a new gene therapy trial could lead to an effective treatment that would involve chemically rebalancing the area of the brain associated with addiction. MONDAY, Aug. With alcohol alone,
In addition to clearing Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy, the FDA also granted an early OK to Bluebird bio’s sickle cell treatment Lyfgenia.
TUESDAY, June 6, 2023 -- Radiation therapy might not be necessary in treating some forms of rectal cancer and lymphoma, sparing patients from the toxic treatment, a pair of new clinical trials shows. One trial found that rectal cancer patients.
MONDAY, April 21, 2025 -- Chris Vogelsang has had a long and terrible fight with cancer. Fourteen years ago, the 70-year-old man was first diagnosed with an aggressive form of lymphoma. His cancer has since returned twice, fighting against several.
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