Search Results for “EC” – Page 373 – media

Genethon’s Gene Therapy for Rare Liver Disease Shows Promising Early-Stage Results

By Connor Lynch Pictured: Illustration of a human body with the liver highlighted/iStock, magicmine Genethon’s experimental gene therapy for treating Crigler-Najjar syndrome, a rare liver disease that causes the toxic buildup of bilirubin, showed promising results in a Phase I/II study published Thursday in The New England Journal of Medicine. The clinical trial, sponsored by Genethon, brought together researchers from Italy, the Netherlands, and France to study the efficacy and safety of Genethon’s GNT 0003 candidate gene therapy. The drug combines an AAV8 vector with a copy of gene UGT1A1, which is defective in patients with the condition. The goal of the treatment is to restore function of the liver and restore its ability to produce the UGT1A1 enzyme, which is responsible for breaking down bilirubin. The study examined 17 women aged 21 to 30 years with severe Crigler-Najjar syndrome. According to the results, the drug restored expression of the ...
- Source: drugdu
- 117
- August 22, 2023
Pfizer, Moderna Target Emerging Variants with Updated COVID-19 Shots

By Tristan Manalac Pictured: Healthcare worker administering a vaccine into a patient’s arm/iStock, Tirachard Moderna on Thursday announced that its updated COVID-19 vaccines can induce neutralizing antibodies against the emerging EG.5 and FL.1.5.1 variants of the virus, dubbed Eris and Fornax, respectively. Pfizer, along with German partner BioNTech, also said on Thursday that its updated shots elicited strong neutralizing activity against the Eris subvariant in a mouse study, reported Reuters. These updated vaccines come just in time for the fall vaccination season, for which the FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) in June unanimously recommended using a monovalent vaccine focusing on the Omicron XBB.1.5 subvariant. According to data from the CDC, XBB.1.5 was the most dominant Omicron subvariant in the U.S. throughout most of 2023, accounting for 73.5% of cases in the latter half of April and 64.5% in the first half of May. In the last ...
- Source: drugdu
- 188
- August 22, 2023
Ipsen’s Sohonos gets FDA approval for fibrodysplasia ossificans progressive

Ipsen’s Sohonos (palovarotene), a drug treatment for fibrodysplasia ossificans progressive (FOP), has been approved by the US Food and Drug Administration (FDA). FOP is a rare muskuloskeletal condition that progressively transforms muscles and tendons into bone, affecting the movement of the body. As a rare disease, FOP is estimated to Impact around 900 people globally. Sohonos has been approved as a treatment for adults and paediatric patients with FOP – for females 8 years and older and for males 10 years and older – to mediate the interactions between the receptors, growth factors and proteins within the retinoid signaling pathway to reduce new abnormal bone formation. The drug has particular selectivity for the gamma subtype of retinoic-acid receptors, which are an important regulator of skeletal development and ectopic bone in the retinoid signaling pathway. “The first treatment for FOP has been proven to reduce the volume of new abnormal bone ...
- Source: drugdu
- 121
- August 22, 2023
Boehringer Ingelheim advances weight loss drug to Phase III trials

Boehringer Ingelheim could soon stake a claim in the lucrative obesity treatment market after the pharma company said it has advanced a candidate into three Phase III trials following promising data. Survodutide, the company’s glucagon/glucagon-like peptide 1 (GLP-1) receptor dual agonist co-developed with Zealand Pharma, demonstrated up to 19% weight loss after 46 weeks of treatment in a Phase II dose escalation trial investigating the drug in patients living with obesity without type 2 diabetes (T2D). In a statement announcing the plans, Boehringer Ingelheim said that insights from previous studies will be used to inform the design of the three Phase III trials which will investigate the efficacy and safety of survodutide. Enrolment of patients is planned before the end of 2023, with the company saying that study details will be unveiled nearer to trial commencement. Survodutide has previously received a US Food and Drug Administration (FDA) fast track designation ...
- Source: drugdu
- 98
- August 22, 2023
AbbVie achieves win for migraine prevention drug Aquipta in Europe

The European Commission has approved AbbVie’s Aquipta as migraine prophylaxis for adult patients, making it the first daily oral treatment of its class to be approved in the EU for the prevention of both chronic and episodic migraines. Several calcitonin gene-related peptide (CGRP) antibodies are administered subcutaneously or via intravenous infusions. But there is a preference among patients for oral therapies, says Dawn Carlson, vice president, Neuroscience Development at AbbVie, in an interview with Pharmaceutical Technology. Lundbeck’s CGRP antibody Vyepti (eptinezumab), is administered through intravenous infusions, while Amgen’s Aimovig (erenumab) is injected subcutaneously. The approval of Aquipta is based on the results of the two Phase III studies—ADVANCE (NCT03777059) and PROGRESS (NCT03855137), which both met their primary endpoint of a statistically significant reduction in mean monthly migraine days versus placebo, per the 17 August announcement. In the PROGRESS trial, patients in the 60mg Aquipta cohort reported a 6.8-day reduction in ...
- Source: drugdu
- 115
- August 22, 2023
【EXPERT Q&A】What is the procedure for registering medical devices for the Russian market?

Drugdu.com expert’s response: The registration process in Russia mainly consists of the following steps: Submission of Application: First, you need to submit a registration application to the Russian Ministry of Health. This typically includes detailed information about your product such as the name, type, specifications, manufacturer information, etc. Technical Documentation Review: The Russian Ministry of Health will review the technical documentation you have submitted. This might encompass details of your product design, production process, quality control systems, etc. Clinical Evaluation: You might be required to undergo a clinical evaluation to demonstrate the safety and efficacy of your product. This could involve clinical trials or submission of other forms of clinical data. Manufacturer Review: The Russian Ministry of Health might review the manufacturer to ensure compliance with Russian laws and regulations. Obtaining the Registration Certificate: If your product passes the above steps of the review, you will receive a registration certificate. ...
- Source: drugdu
- 321
- August 22, 2023
medical device Russia market
Meta-analysis reports global prevalence of MAFLD of 38.8% in adults

Non-alcoholic fatty liver disease (NAFLD) is a common condition that affects up to 30% of adults in the general population. It is characterized by the accumulation of fat in the liver. It is often associated with other conditions, such as obesity, type 2 diabetes, and metabolic syndrome. The new definition of metabolic dysfunction-associated fatty liver disease (MAFLD) was proposed in 2020. MAFLD is diagnosed based on hepatic steatosis (fatty liver) and at least one of three metabolic risk abnormalities: overweight/obesity, type 2 diabetes, or evidence of metabolic dysregulation. A recent meta-analysis led by Ming-Hua Zheng from the First Affiliated Hospital of Wenzhou Medical University has reported a global prevalence of MAFLD of 38.8% in adults. MAFLD can progress to cirrhosis and promote the development of some critical extrahepatic diseases, such as cardiovascular disease and chronic kidney disease. MAFLD is a severe condition that can lead to cirrhosis and other health ...
- Source: drugdu
- 181
- August 21, 2023
Severe COVID-19 may cause long-term changes to innate immune system

Severe COVID-19 may cause long-lasting alterations to the innate immune system, the first line of defense against pathogens, according to a small study funded by the National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health. These changes may help explain why the disease can damage so many different organs and why some people with long COVID have high levels of inflammation throughout the body. The findings were published online today in the journal Cell. Researchers led by Steven Z. Josefowicz, Ph.D., of Weill Cornell Medicine in New York City examined immune cells and molecules in blood samples from 38 people recovering from severe COVID-19 and other severe illnesses, as well as from 19 healthy people. Notably, the researchers established a new technique for collecting, concentrating and characterizing very rare blood-forming stem cells that circulate in the blood, eliminating the need to extract such cells from ...
- Source: drugdu
- 177
- August 21, 2023
Chimerix’s Drug Candidate Improves Survival for Patients with Incurable Brain Tumor

By Matt Olszewski Pictured: Adult holding child’s hand/gorodenkoff/iStock Biopharma company Chimerix announced Wednesday positive results from two early-stage clinical trials for its first in-class small molecule imipridone, a potential treatment for an incurable and aggressive type of pediatric brain tumor which demonstrated longer survival in this subset of patients. The data, published Wednesday in the journal Cancer Discovery, showed that Chimerix’s ONC201 nearly doubled survival for patients with H3K27M-mutated diffuse midline gliomas (DMG), as compared to previous patients. Diffuse midline gliomas with the H3K27M mutation are aggressive, with a survival rate of 11 months to 15 months. However, the median overall survival was nearly 22 months for tumors that had not recurred at the time of enrollment in the trial. In addition, almost a third of those patients lived longer than two years. “These data further elucidate the underlying novel mechanism of action for ONC201 in a patient population which ...
- Source: drugdu
- 114
- August 21, 2023
FDA Approves Ipsen’s Palovarotene for Ultra-Rare Bone Disease

By Tristan Manalac Pictured: U.S. FDA headquarters in Maryland/iStock, Grandbrothers The FDA on Wednesday approved Ipsen’s palovarotene, now to be sold under the brand name Sohonos, for the treatment of fibrodysplasia ossificans progressiva. Sohonos is an oral medication indicated for the reduction of heterotrophic ossification in adults and children with fibrodysplasia ossificans progressiva (FOP). It is the first medicine authorized by the FDA to treat FOP. “For the first time, doctors have an approved medicine available to them, shown to reduce the formation of new, abnormal bone growth,” Ipsen R&D head Howard Mayer said in a statement. Afflicting approximately 400 patients in the U.S. and 900 globally, FOP is an ultra-rare genetic disease in which muscles and connective tissues are gradually replaced by bone. Patients with FOP often suffer from hindered movements and, in cases where joints are affected, lose their mobility. FOP also manifests as breathing problems and difficulties ...
- Source: drugdu
- 108
- August 21, 2023

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