Roche has announced positive new four-year results from an open-label extension study of its spinal muscular atrophy (SMA) treatment, Evrysdi (risdiplam), in young children. SMA is a severe and progressive neuromuscular disease affecting approximately one in every 10,000 babies. People living with the condition have insufficient levels of the SMN protein, which is essential to the function of nerves that control muscles and other functions such as swallowing, speaking, breathing and movement. Depending on the form of SMA, the patient’s physical strength and their ability to walk, eat or breathe can be considerably compromised or lost. Without treatment, children with type 1 SMA are not expected to live past age two and are never able to sit without support. Roche’s Evrysdi, which can be administered at home in liquid form by mouth or by feeding tube, is designed to treat the disease by increasing and sustaining the production of SMN ...
A group led by researchers at Nagoya University in Japan has developed a technology to capture and release cell-free DNA (cfDNA) on nanowire surfaces from urine. By extracting this DNA, they were able to successfully detect IDH1 mutation, a characteristic genetic mutation of gliomas, a type of brain tumor. Their findings increase the effectiveness of cancer detection tests using urine. They published their results in the journal Biosensors and Bioelectronics. Brain tumors are often examined only after the appearance of symptoms, such as paralysis of the limbs. But even when they are detected, they are often so advanced that it is difficult to remove them by surgery. Among these tumors, some of the deadliest are gliomas. These tumors have an average survival time as low as 12-18 months. Therefore, for the patient to have a chance of survival, early detection is necessary. As many patients have routine physicals in which ...
Motor vehicles are among the leading causes of death by injury, with the World Health Organization (WHO) reporting that road traffic accidents comprised over a fifth of all fatal injuries in 2015. Across the world, over 30,000 people suffer serious injuries from such accidents every day. Electric scooters (e-scooters) have been linked to an increased risk of harm. A new research paper published in the journal JAMA Network Open looks at the impact of nighttime speed limits on injury risk related to e-scooters. Introduction Since their introduction in many countries, e-scooters have been associated with high injury statistics, especially to the head and face. In particular, nighttime driving of e-scooters, or driving under the influence of alcohol or illegal substances, has been found to be associated with injury risk in a substantial proportion of cases. In attempts to reduce the toll from road traffic accidents, the safe system approach has ...
Australia on Saturday became one of the first countries in the world to allow the use of MDMA and magic mushrooms for medical treatment, in a bid to tackle certain mental health conditions. From July 1, authorized psychiatrists will be able to prescribe the drugs, also known as ecstasy and psilocybin, for the treatment of post-traumatic stress disorder and some types of depression. Authorities in Canada and the United States allow the medical use of one or both of the drugs, but only in clinical trials or with special permits. In February, Australia reclassified the drug entirely, after the country’s Therapeutic Goods Administration said trials had found the substances to be “relatively safe” when used in a “medically-controlled environment”. Supporters of the move hope the drugs can provide breakthroughs for mental health patients, when other treatments have failed. Mike Musker, a mental health and suicide prevention researcher at the University ...
Florida International University scientists discovered the first and only known natural arsenic-containing antibiotic to fight antibiotic resistance. Now, research reveals it can stop transmission of a deadly disease spreading in the U.S. for the first time in 20 years: malaria. A team from FIU’s Herbert Wertheim College of Medicine developed arsinothricin (AST) to combat the rise of antibiotic-resistant bacteria. Lab tests proved AST effectively defeated the most notorious, including E. coli and Mycobacteria, which cause tuberculosis. Collaborating with malaria researchers in the College of Arts, Sciences & Education, they’ve recently also found AST prevents Plasmodium falciparum, the parasite that causes malaria, from infecting mosquitoes—unlike other current antimalarial drugs. The discovery, recently published in Microorganisms, paves the way for AST to one day be developed into a more effective antimalarial drug for humans. “Current antimalarials don’t completely stop transmission, meaning patients can continue to infect mosquitoes before they recover,” said lead ...
by Danielle Ellis, B.Sc. A group led by researchers at Nagoya University in Japan has developed a technology to capture and release cell-free DNA (cfDNA) on nanowire surfaces from urine. By extracting this DNA, they were able to successfully detect IDH1 mutation, a characteristic genetic mutation of gliomas, a type of brain tumor. Their findings increase the effectiveness of cancer detection tests using urine. They published their results in the journal Biosensors and Bioelectronics. Brain tumors are often examined only after the appearance of symptoms, such as paralysis of the limbs. But even when they are detected, they are often so advanced that it is difficult to remove them by surgery. Among these tumors, some of the deadliest are gliomas. These tumors have an average survival time as low as 12-18 months. Therefore, for the patient to have a chance of survival, early detection is necessary. As many patients have ...
By Tristan Manalac Pictured: BioMarin headquarters/iStock, Michael Vi The FDA has approved BioMarin Pharmaceutical’s valoctocogene roxaparvovec-rvox, to be marketed as Roctavian in the U.S., for the treatment of adult patients with severe hemophilia A, the company announced Thursday. Roctavian, a one-time single-dose infusion, is authorized for use only in adults without antibodies against the adeno-associated virus serotype 5 (AAV5), as determined by an FDA-approved test. It is also the first gene therapy approved for hemophilia A, according to BioMarin’s news release. In an investor call Thursday afternoon, BioMarin Chief Commercial Officer Jeff Ajer said that “the product profile of Roctavian presents tremendous value to patients, including bleed control, good safety profile and freedom from the burden of chronic therapy.” Roctavian is “priced at a wholesale acquisition cost, or WAC, that equates to $2.9 million for the typical patient for this one-time, single-dose treatment,” Ajer said. Hemophilia A is an inherited ...
The first drug fully generated by artificial intelligence entered clinical trials with human patients this week. Insilico Medicine, a Hong Kong-based biotech startup with more than $400 million in funding, created the drug, INS018_055, as a treatment for idiopathic pulmonary fibrosis, a chronic disease that causes scarring in the lungs. The condition, which has increased in prevalence in recent decades, currently affects about 100,000 people in the U.S. and can lead to death within two to five years if untreated, according to the National Institutes of Health. “It is the first fully generative AI drug to reach human clinical trials, and specifically Phase II trials with patients,” Alex Zhavoronkov, founder and CEO of Insilico Medicine, told CNBC. “While there are other AI-designed drugs in trials, ours is the first drug with both a novel AI-discovered target and a novel AI-generated design.” The discovery process for the new drug began in ...
The US Food and Drug Administration (FDA) has approved CellTrans’ Lantidra (donislecel) as the first cellular therapy to treat patients with type 1 diabetes. The authorisation specifically applies to adults who are unable to meet their target blood glucose levels because they have repeated episodes of severe low blood sugar (hypoglycaemia), despite intensive disease management and education. Almost 1.9 million people in the US have type 1 diabetes, according to the American Diabetes Association. The condition requires lifelong care, including the regular administration of insulin, either through multiple daily injections or continuous infusion using a pump. However, some patients have trouble managing the amount of insulin needed every day, and dosing becomes difficult. These patients may also develop hypoglycaemia unawareness, where they are unable to detect that their blood glucose is dropping and may not have a chance to treat themselves. Lantidra, which is administered initially as a single infusion, ...
The US Food and Drug Administration (FDA) has announced that Lantidra, a cellular therapy for type 1 diabetes (T1D), has become the first treatment of its kind to be approved. Lantidra is an allogeneic (donor) pancreatic islet cellular therapy made from deceased donor pancreatic cells. The approval applies to adults with ‘brittle’ T1D – those unable to reach target glycated haemoglobin (average blood glucose levels) due to repeated severe hypoglycaemic episodes, despite intensive diabetes management and education. T1D accounts for roughly 5%-10% of all diabetes cases. Within this population, an even smaller group, approximately three out of every 1,000 people with T1D, suffer from ‘brittle’ disease. The therapy works by essentially replacing the body’s insulin-producing beta cells in the pancreas. This is achieved through allogeneic islet beta cells that secrete insulin, administered as a single infusion into the hepatic (liver) portal vein. While this is generally sufficient, an additional infusion ...
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