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Study reveals unique molecular machinery of woman who can’t feel pain

The biology underpinning a rare genetic mutation that allows its carrier to live virtually pain-free, heal more rapidly and experience reduced anxiety and fear, has been uncovered by new research from UCL. The study, published in Brain, follows up the team’s discovery in 2019 of the FAAH-OUT gene and the rare mutations that cause Jo Cameron to feel virtually no pain and never feel anxious or afraid. The new research describes how the mutation in FAAH-OUT “turns down” FAAH gene expression, as well as the knock-on effects on other molecular pathways linked to wound healing and mood. It is hoped the findings will lead to new drug targets and open up new avenues of research in these areas. Jo, who lives in Scotland, was first referred to pain geneticists at UCL in 2013, after her doctor noticed that she experienced no pain after major surgeries on her hip and hand. ...
- Source: drugdu
- 101
- May 25, 2023
The power of genetics — how research into gene sequencing could revolutionize biopharma

Unlocking the potential of human genetics is key to many advances in the biopharma industry. From immunotherapies that treat cancer to potential cures for rare diseases to mapping the genome for incredible medical discoveries, researchers have still only scratched the surface of what is possible in gene science. Just last week, the FDA approved the first-ever gene therapy that can be applied directly to the skin to treat patients known as “butterfly children” because of how fragile their skin can be. These kinds of forward-thinking medicines have come about due to the ever more advanced understanding of how genetics cause certain conditions. And early research in the genetic space — even that which might not appear directly related to medicine — has given scientists the tools to make better treatments down the road. Some of those treatments are on their way to helping patients soon. One cell therapy in late-stage ...
- Source: drugdu
- 118
- May 25, 2023
Crick Institute announces new molecular glue discovery to treat different diseases

A combined investment of £11.2m that includes a prosperity partnership grant from the Engineering and Physical Sciences Research Council (EPSRC) and funds from AstraZeneca (AZ) has enabled researchers at the Francis Crick Institute, Imperial College London and AZ to discover new ‘molecular glues’ to treat multiple diseases. Widely used as protein degraders, molecular glues bring together proteins that would not usually interrelate. The glues manage the modification and degradation of disease-causing target proteins by redirecting them to the cell’s natural waste disposal system. By facilitating the interaction between target protein and a cellular enzyme, the target protein is deactivated or broken down by additional proteins that are recruited by the enzyme. Group leader at the Crick Institute, Ed Tate, said: “Challenges arise when we can’t find a small molecule with the ability to interact with certain proteins. “If we can redirect these harmful proteins towards the cell’s own waste disposal ...
- Source: drugdu
- 115
- May 25, 2023
A larger dose of vitamin D may decrease psychiatric symptoms at school age

It is estimated that every eighth child suffers from a mental health disorder. Several predictors of children’s mental problems have been identified, but much remains uncharted. Previous research suggests that low vitamin D levels in early childhood may be one factor increasing the risk of mental health problems in later life. A recent Finnish study, now published in JAMA Network Open, presents new information on the association between vitamin D intake and mental health. A new study by a team of Finnish researchers has investigated whether a daily vitamin D3 supplement higher than the recommended dose in early childhood reduces the risk of psychiatric symptoms at school age. The study is part of the Vitamin D Intervention in Infants (VIDI) clinical trial, which investigates how early vitamin D3 intervention affects children’s growth and development. In the research, children were randomized as two groups, one receiving the daily standard dose of ...
- Source: drugdu
- 143
- May 25, 2023
Amgen, J&J ink patent settlement

After the recent high-profile loss of exclusivity for AbbVie’s megablockbuster immunology drug Humira, industry watchers’ eyes moved to Johnson & Johnson’s Stelara as one of the next major drugs expected to face biosim competition. Tuesday, the industry got more information about when exactly that biosimilar clash might occur. In J&J’s patent case over Amgen’s proposed biosimilar to J&J’s Stelara, the companies have settled, according to a Tuesday filing in Delaware federal court. An Amgen spokesperson told Reuters the deal will allow the company to launch its Stelara biosimilar “no later than January 1st, 2025.” With that, the market exclusivity clock is ticking on J&J’s biggest drug by sales. Last year, the medicine pulled down $9.72 billion across its range of indications, including plaque psoriasis, psoriatic arthritis and Crohn’s disease. Amgen has yet to win FDA approval for its proposed biosimilar. The company filed its biosim application last November, so the ...
- Source: drugdu
- 106
- May 25, 2023
Ironwood to Pay $1B to Acquire VectivBio, Late-Stage Digestive Therapy

By Jef Akst Pictured: Illustration of a human body with the intestines highlighted/iStock, Rasi Bhadramani At $17 per share, Ironwood Pharmaceuticals has agreed to buy VectivBio for around $1 billion in a cash deal. In doing so, the Boston-based company, which already markets Linzess for irritable bowel syndrome and constipation with AbbVie, will expand its gastrointestinal drug portfolio. The Swiss biotech’s most advanced product is apraglutide, being developed for short bowel syndrome with intestinal failure (SBS-IF) that affects some 18,000 adult patients in the U.S., Europe and Japan, according to the Ironwood press release. People with the disorder are unable to absorb adequate nutrition from their food, leading to impaired quality of life and sometimes death. Takeda Pharmaceuticals has an approved drug, Gattex, that aims to treat short bowel syndrome but requires daily injections. Apraglutide is being tested in a Phase III study involving weekly injections. “There’s certainly challenges with ...
- Source: drugdu
- 139
- May 24, 2023
Pfizer oral weight loss drug may be as effective as Ozempic injection by Novo Nordisk, study says

An oral drug made by Pfizer causes a similar amount of weight loss as rival Novo Nordisk’s blockbuster injection Ozempic, according to a peer-reviewed study of phase 2 clinical trial results released Monday. The results were presented at a medical conference late last year, and did not compare Pfizer’s drug with Ozempic or other weight loss medications. JAMA Network only now is releasing a peer-reviewed study. Pfizer’s trial followed 411 adults with Type 2 diabetes who either took the company’s pill, danuglipron, twice a day or a placebo. Body weight was “statistically significantly reduced” after patients took either 120-milligram or 80-milligram versions of danuglipron for 16 weeks, the study found. Patients who took a 120-milligram version lost around 10 pounds on average over that time period, the study found. Pfizer’s drug could offer an advantage as an oral treatment option rather than a frequent injection. The study results also suggest ...
- Source: drugdu
- 151
- May 24, 2023
Beta Bionics wins FDA nod to challenge Medtronic for automated insulin pump market

Dive Brief: Beta Bionics said it received 510(k) clearance from the Food and Drug Administration for its automated insulin dosing (AID) system, teeing it up to challenge Insulet, Medtronic and Tandem Diabetes Care for the Type 1 diabetes market. The system, iLet Bionic Pancreas, features an insulin pump and dosing software that combine with the Dexcom G6 continuous glucose monitor to automate the calculation and delivery of insulin doses. Beta Bionics, which showed the AID system beat standard of care last year, has differentiated its device through simplification and automation, limiting the information users need to input and preventing them from modifying their insulin doses. Dive Insight: The launch of iLet Bionic Pancreas may serve as a test of whether people with Type 1 diabetes are willing to almost totally cede control of their insulin dosing to an algorithm. Users of existing hybrid closed-loop systems enter information such as basal ...
- Source: drugdu
- 117
- May 24, 2023
A clever way to detect narcissism

A team of psychologists at the University of Helsinki, working with a colleague from Millsaps College, has discovered, via experimentation, a clever, new way to detect narcissism in a person. In their study, reported in the journal Psychophysiology, the group measured physical responses to certain stimuli in people identified as having a narcissistic personality. People with a narcissistic personality see the world as an extension of themselves, and because of that, they regard the needs of others as less important. Narcissists, it has been noted, tend to be stubborn and envious and behave in ways that suggest they feel entitled to whatever they desire. Psychologists have also found that such traits can make a person uncomfortable to be around and that others tend to respond negatively to such behavior. They also note that narcissistic traits can be problematic for relationships, and for that reason, they have suggested that non-narcissists learn ...
- Source: drugdu
- 123
- May 24, 2023
AbbVie and Genmab’s blood cancer therapy granted FDA accelerated approval

AbbVie and Genmab’s blood cancer therapy, Epkinly (epcoritamab-bysp), has been granted accelerated approval by the US Food and Drug Administration (FDA). The authorisation specifically applies to adults with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) who have received at least two prior lines of treatment. DLBCL is the most common type of aggressive lymphoma, with approximately 150,000 cases of the disease diagnosed globally each year. For R/R patients, several targeted therapies including T-cell mediated treatments have recently emerged, the companies said. However, single agent and ready-available or off-the-shelf treatment options are limited. Epkinly, which is being co-developed by AbbVie and Genmab as part of the companies’ oncology collaboration, is now the first and only T-cell engaging bispecific antibody for this indication. The FDA’s decision was supported by positive results from a phase 1/2 trial, in which Epkinly delivered an overall response rate of 61%, a complete response rate ...
- Source: drugdu
- 117
- May 24, 2023

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