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FDA Extends Trikafta Approval Even to Young Cystic Fibrosis Patients



The FDA has expanded indications for the use of elexacaftor, tezacaftor, and ivacaftor (trikaft) for cystic fibrosis to include even young children. Vertex Pharmaceuticals announced..

Three drug combination extended shortcut currently covers children aged 2 to 5 years with cystic fibrosis and F508del mutation in CFTR gene or CFTR a gene mutation that has been shown to respond to a triplet based on in vitro data.

FDA approval for this indication was upheld. phase III open trial this showed an improvement in sweat chloride concentration and lung function in this young population after 24 weeks of treatment, with a safety profile consistent with that observed in older age groups.

“Early intervention with a CFTR modulatory therapy such as Trikafta may offer the potential to improve trajectory [cystic fibrosis] lung disease,” said researcher Jennifer Goralsky, MD, of the University of North Carolina School of Medicine at Chapel Hill. cause of their illness.

As of 2022 about 40,000 people in the USA living with cystic fibrosis.

Based on the results of two randomized trials, the triple combination won first. OK in 2019 for adolescents and adults with susceptible CFTR Gene mutations, a group representing approximately 90% of the CF patient population at the time. The subsequent expansion of the label allowed its use in patients aged 6 years and older.

The labeling of elexacaftor, tezacaftor and ivacaftor warns of increased transaminase activity, liver damage and cataracts. In addition, concomitant use with strong CYP3A inducers may reduce the exposure and efficacy of the triple drug.

The product is available in oral granules or tablets, depending on the age and weight of the patient, and should be taken with a fat-containing meal. Patients are advised to avoid eating or drinking food or drinks containing grapefruit while taking triplet therapy.

  • Elizabeth Short is a staff writer for MedPage Today. She is often involved in pulmonology, allergology and immunology. Follow

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Tablets that destroy stomach cells will help to cope with nausea and lack of appetite



The swallowed capsule is covered with grooves that drain liquid.

Jay McRae (MIT)

The tablet has been used to electrically stimulate stomach cells in pigs to increase levels of ghrelin, a hormone that regulates hunger and relieves nausea. If the technology is transferred to humans, it could treat nausea, vomiting, and lack of appetite in people with eating disorders or those being treated for cancer.

Current interventions using electrical stimulation to relieve gastrointestinal symptoms require invasive surgery. So, Giovanni Traverso at the Massachusetts Institute of Technology and colleagues have developed a non-invasive, growth-sized capsule that delivers small shocks.

Inside the capsule, a battery generates electrical currents that run through gold wire wound on the outside. Grooves etched into the surface of the capsule drain fluid so that the wire can electrically stimulate receptors in the stomach tissue that trigger the release of ghrelin. The device is coated with a thin shell that dissolves when it enters the stomach so as not to irritate the tissues of the esophagus.

Traverso and his team tested the capsule on 13 pigs that were fasted overnight and given anesthetics. Eleven animals received a 20-minute stimulation and two others received no stimulation.

The researchers measured blood levels of ghrelin before and 10 minutes after the stimulation. They found that, on average, ghrelin levels increased by about 40% in stimulated pigs, while ghrelin levels decreased by about 50% in unstimulated pigs.

“The levels we are seeing are comparable to the levels we would expect to induce hunger or suppress nausea, but we have not evaluated these levels. [symptoms] in animals,” says Traverso. “Part of the next steps involves doing this in humans to see if, for example, nausea can be treated with this system.”

Traverso said all animals cleared the capsule within two weeks, a normal time for pigs. Tissue samples taken from their stomachs, duodenums and colons showed no change or signs of injury, suggesting the therapy was safe.

“To my knowledge, this is the first ingestible device that can produce electrical stimulation of the gastrointestinal tract,” he says. Stavros Skid at the Feinstein Institute for Medical Research in New York. The technology can be modified to provide electrical stimulation to other tissues in the gastrointestinal tract, such as the colon.


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Overview: Seoul National University Hospital promotes AI-assisted early diagnosis of autism and other updates



Seoul National University Hospital Opens Living Lab for Early Diagnosis of Autism

The Seoul National University Hospital in South Korea has opened a living lab that aims to promote early diagnosis and personalized treatment of autism spectrum disorders.

The lab, comprised of four rooms: interaction rooms, eye tracking rooms, observation rooms, and family consultation rooms, will collect live health data on children with autism, such as voice, language, and gaze.

This data will be used to develop artificial intelligence models for early diagnosis and personalized treatment of autism.

The laboratory, established as part of a project by the National Center for Mental Health under the Ministry of Health and Welfare, will also serve as the basis for various digital treatments and the discovery of autism biomarkers.

Aster DM Healthcare launches digital health center in India

Aster DM Healthcare has opened a telecommand center and digital health office in Bangalore, India.

According to a press release, the center allows a team of qualified medical professionals to monitor and treat patients remotely in real time. It also serves as the basis for a number of hospital telemedicine operations in India, including virtual consultations, remote patient monitoring, tele-ICT, teleradiology and other telemedicine-based interventions.

“With this new initiative, we are now able to bring the experience of our talented healthcare professionals beyond physical boundaries to patients and professionals around the world,” said Dr. Azad Mupen, Founding Chairman and Managing Director of Aster DM.

Meanwhile, Aster DM will also soon be launching myAster, its omnichannel healthcare offering, in India.

South Korean partnership to use AI to build knowledge base on rare diseases

SNUH also recently announced a partnership with the Mogama Biomedical Research Institute to build a rare disease knowledge base using AI.

According to a press statement, the knowledge base will help collect and interpret information about the causes, symptoms, and genetics of rare diseases for diagnosis and treatment.

The Mogama Institute will develop AI algorithms to create said knowledge base, and SNUH will review the available data.

Saitama Medical University Hospital to Test AI for Migraine Diagnosis

Saitama Medical University Hospital in Japan has teamed up with US-based artificial intelligence company Healint to conduct the world’s first clinical trial on AI-assisted headache diagnosis.

The study, according to a press statement, aims to test the AI ​​in the Healint Migraine Buddy app to detect migraine patterns.

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Ms Manners: Children’s gesture of empathy is prohibited by school rules



Dear Miss Manners: My 6 year old daughter has had a best friend ever since the girls were old enough to notice another little person sitting next to them in a stroller. During covid, her mom and I had outdoor games and video calls. Girls are like sisters.

Shortly before they were due to start kindergarten last year, their friend “Jenna” was diagnosed with a form of cancer with a very low survival rate. My daughter understands that Jenna has a terrible disease and that the doctors will do everything possible, but her friend may have to go to heaven. For the most part, she handles the situation just fine.

But there is also a hat. My daughter decided that she and Jenna needed matching hats when Jenna’s hair started to fall out so it was a “fashion statement” and not a problem. She insists on putting her hair in a bun and covering it with a hat so that she and her friend match. Over time, the hat seemed to become an object of security, as well as a bond with her friend, who couldn’t go to school and was often too sick to play. My girl is still small and trying to handle really big things. I understand that she needs it. But her teacher and school are not.

I understand that small children lose subtlety and rules should be rules, so I tried to compromise with the school no-hat rule: can she keep her hat with her but just not wear it indoors? Other kids brought soft toys for comfort, so why not a hat?

The teacher was not happy with this, and she called me more than once to chastise me and call my daughter “naughty.” She even removed my little girl’s hair for school photos, resulting in a collection of photos of her sobbing.

Yes, the teacher does not like the thought of the death of a child. Who is not? But there’s my little girl, who has far less maturity and understanding to help her cope, and she feels like she’s being picked on when she needs support. I need Miss Manners’ decision on the hat and any advice on how best to proceed in this situation.

While immensely touched judging by your daughter’s empathy, Miss Manners also has some sympathy for a teacher who doesn’t want to break the rules for one child. But it’s clear that this teacher has problems far beyond that, not least touching your daughter’s hair.

An immediate solution to the hat problem might be to put it on top of a stuffed animal when she comes to school. But if that teacher still won’t give in, Ms. Manners suggests that you ask the principal to intervene.

Surely your daughter is not the first student and not the last to experience loss. Whatever the school’s underlying problem, administrators must explain their policy or find their own sensitive way of dealing with it.

New Miss Manners columns are published Monday through Saturday at You can send questions to Miss Manners on her website, You can also follow her @RealMissManners.

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