Babies born with infantile-onset Pompe disease typically have enlarged hearts and weak muscles. But 1-year-old Ayla has a normal heart and walks.
Category: biotech/medical – Page 1,121
If QPX9003 makes it through Phase II and III clinical trials and onto the market, it will be the first new lipopeptide antibiotic that targets Gram-negative bacteria in over 60 years.
QPX9003, a new antibiotic for Gram-negative bacteria, has achieved success in a Phase I clinical trial.
Similar to a mouse racing through a maze, making “yes” or “no” decisions at every intersection, researchers have developed a way for machines to swiftly learn all the twists and turns in a complex data system.
“Our method may help improve the diagnosis of urinary diseases, the imaging of cardiac conditions and analysis of financial risks,” reported Abd-AlRahman Rasheed AlMomani of Embry-Riddle Aeronautical University’s Prescott, Arizona, campus.
The research was accepted for the Nov. 11 edition of the journal Patterns with Jie Sun and Erik Bollt of Clarkson University’s Center for Complex Systems Science. The goal of the work is to more efficiently analyze binary (“Boolean”) data.
ISRIB, a tiny molecule identified by University of California, San Francisco (UCSF) researchers can repair the neural and cognitive effects of concussion in mice weeks after the damage, according to a new study.
ISRIB blocks the integrated stress response (ISR), a quality control process for protein production that, when activated chronically, can be harmful to cells.
The study, which was recently published in the Proceedings of the National Academy of Sciences, discovered that ISRIB reverses the effects of traumatic brain injury (TBI) on dendritic spines, an area of neurons vital to cognition. The drug-treated mice also showed sustained improvements in working memory.
A toddler is thriving after doctors in the U.S. and Canada used a novel technique to treat her before she was born for a rare genetic disease that caused the deaths of two of her sisters.
Ayla Bashir, a 16-month-old from Ottawa, Ontario, is the first child treated as a fetus for Pompe disease, an inherited and often fatal disorder in which the body fails to make some or all of a crucial protein.
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Gene therapies can scale economically, but not just with practices adapted from traditional biologics. According to Avantor, gene therapies pose unique material, workflow, and partnering challenges.
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For decades, scientists have sought to create red blood cells in the lab – a “holy grail” that some hoped could ease regional blood shortages, especially for people with rare blood types.
But now British researchers say they have overcome a major barrier that has plagued many scientists: creating enough red cells to fill a blood bag. Their findings are published in the journal Nature Communications.
“When we kept (the cells) continually dividing for a year, we were quite excited,” said Jan Frayne, a biochemist at the University of Bristol and one of the study’s lead authors.
Scientists at the University of Pittsburgh School of Medicine have discovered the missing puzzle piece in the mystery of how melanoma tumors control their mortality.
In a paper published in Science this week, Jonathan Alder, Ph.D. and his team describe how they discovered the perfect combination of genetic alterations that tumors use to promote explosive growth and prevent their own demise, a development that could change the way oncologists understand and treat melanoma.
“We did something that was, in essence, obvious based on previous basic research and connected back to something that is happening in patients,” said Alder, assistant professor in the Division of Pulmonary, Allergy and Critical Care Medicine at Pitt’s School of Medicine.
Insilico Medicine is radiating heat amid the biotech winter, kindling its fires with a Sanofi collaboration that could be worth up to $1.2 billion in biobucks—the AI drug discovery company’s larges | Insilico Medicine is radiating heat amid the biotech winter, kindling its fires with a Sanofi collab that could be worth up to $1.2 billion in biobucks—the AI drug discovery company’s largest deal to date.
Recently, two patients in the United Kingdom received two small doses of lab-grown blood samples as part of the RESTORE trial.
Image Credit: Sashkin / Shutterstock.com
About the RESTORE trial
The RESTORE trial is a single-center, randomized, controlled, phase I cross-over trial that is a joint effort between the National Health Service (NHS) Blood and Transplant (NHSBT) and the University of Bristol. The aim of this trial is to determine whether blood cells manufactured from donor stem cells perform better than red blood cells (RBCs) obtained from the same donor in recipients.