Taysha Gene Therapies Receives Rare Pediatric Disease Designation and Orphan Drug Designation for TSHA-102 as a Treatment for Rett Syndrome

Program leverages novel miRARE platform technology used to control transgene expression on a cellular basis

TSHA-102 anticipated to submit Investigational New Drug application in 2021

Taysha Gene Therapies Inc. (Nasdaq: TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced that it has received rare pediatric disease designation and orphan drug designation from the U.S. Food and Drug Administration (FDA) for TSHA-102, an AAV9-based gene therapy in development for the treatment of Rett syndrome. Taysha anticipates that it will submit an Investigational New Drug (IND) application for TSHA-102 to the FDA in 2021.

Rett syndrome is one of the most common genetic causes of severe intellectual disability worldwide, with a prevalence of over 25,000 cases in the U.S. and European Union (EU). It is an X-linked disease that primarily occurs in females, but it can be seen very rarely in males. It is usually recognized in children between six to 18 months of age as they begin to miss developmental milestones or lose abilities they had developed. Individuals with Rett syndrome also show symptoms that include loss of speech, loss of purposeful use of hands, loss of mobility, seizures, cardiac impairments, breathing issues and sleep disturbances.

“Patients with Rett syndrome are currently managed with symptomatic treatments as there are no therapies approved to treat the underlying cause of disease,” said Berge Minassian, M.D., Chief Medical Advisor of Taysha and Chief of Pediatric Neurology at the University of Texas Southwestern Medical Center (UT Southwestern). Dr. Minassian is credited with describing the CNS isoform of the MECP2 gene which is responsible for neuronal and synaptic function throughout the brain. “Gene therapy offers a potentially curative option for patients suffering with Rett syndrome.”

Rett syndrome is caused by mutations in the MECP2 gene. TSHA-102 is designed to deliver a healthy version of the MECP2 gene as well as the miRNA-Responsive Auto-Regulatory Element, miRARE, platform technology to control the level of MECP2 expression. “TSHA-102 represents an important step forward in the field of gene therapy, where we are leveraging a novel regulatory platform called miRARE to prevent the overexpression of MECP2,” said Steven Gray, Ph.D., Chief Scientific Advisor of Taysha and Associate Professor in the Department of Pediatrics at UT Southwestern. “In collaboration with Sarah Sinnett, Ph.D. to develop miRARE, our goal was to design a regulated construct that allowed us to control MECP2 expression to potentially avoid adverse events that are typically seen with unregulated gene therapies.”

The FDA defines a rare pediatric disease as a serious or life-threatening disease in which the disease manifestations primarily affect individuals aged from birth to 18 years. Pediatric diseases recognized as “rare” affect under 200,000 people in the U.S. The Rare Pediatric Disease Priority Review Voucher Program is intended to address the challenges that drug companies face when developing treatments for these unique patient populations. Under this program, companies are

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Diamond Dental Associates LLC, Has The Best Flemington Dentist For Oral Healthcare And Treatment Needs

Diamond Dental Associates LLC, Has The Best Flemington Dentist For Oral Healthcare And Treatment Needs

Flemington, NJ – Members of the Flemington, NJ community who wish to sport the best smiles and enjoy the advantages of top oral health can rest assured that Diamond Dental Associates LLC has the best dentists in the area to deliver these premium services at the most affordable prices. At Diamond Dental Associates LLC, the dental team focuses on providing premium quality dental care and treatment solutions to patients while delivering the best dental experience in the process.

As the leading dental clinic in the area, Diamond Dental Associates LLC has a team of dentists with over three decades of experience who are on the ground to listen to oral health complaints from patients and address the problem. At Diamond Dental Associates LLC, the team employs the use of the latest dental technologies to establish diagnosis and craft treatment plans that are most suitable for the needs of each patient.

Promising the very best dental experience, the top Flemington Dentist at the clinic said: “Here at Diamond Dental Associates LLC, we pledge to provide the finest personal service using state of the art technology for our patients, who will always be welcomed to a warm and caring environment. At Diamond Dental Associates we change lives one smile at a time. We are committed to making every interaction with our patients a positive one. We respect and acknowledge your concerns about the dental treatment you will receive. We recognize that it is difficult for highly fearful people to visit a dental office for needed treatment and we offer services that help you to feel confident that the treatment we provide is in your best interests and fits in with your preferences, lifestyle, and monetary investment.”

As a dental clinic that is constantly working on new ways to address the dental care and treatment needs of patients, Diamond Dental Associates LLC – Flemington Dentist has expanded the services offered to include general dentistry practice which focuses on improving oral health and protecting the mouth from common debilitating conditions. As part of the general dentistry services offered, patients will get to enjoy routine dental examinations, oral cancer screening, teeth cleaning, dental sealants, and more.

Added to this, the dental team at Diamond Dental Associates LLC also offers a wide range of cosmetic dental solutions aimed at improving the appearance and appeal of the teeth. As part of the cosmetic dental solutions offered, patients are welcomed into the clinic to enjoy teeth whitening services aimed at brightening the teeth and smile; cosmetic bonding used to restore decayed teeth, strengthen a weak tooth, close spaces between the teeth, and alter the shape and/or size of the tooth.

The dentists also offer dental veneers that are used to cover up blemishes, chips, cracks, and tooth discoloration; dental implants for restoration of missing or lost teeth; as well as sports mouthguards and sleep apnea treatment.

Visit Diamond Dental Associates LLC at North, 334 NJ-31 #1, Flemington, NJ 08822, or call 908-838-0004. For more information, send an email

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Doctor on frontline issues warning for COVID-19 treatment: ‘This medicine … will surely kill them’

Dr. Tom Yadegar, a specialist in critical care medicine who has been treating some of the most extreme cases of the coronavirus, joined Glenn Beck on the radio program Tuesday to share some important information with the public and doctors around the world about treating COVID-19.

As the intensive care unit director at Providence Cedars-Sinai Tarzana Medical Center in California, Yadegar and has been treating patients with COVID-19 for about seven weeks and has not lost a single patient. He was recently asked to command four more regional hospitals because he and his team are performing well above other hospitals in the Los Angeles area.

Yadegar created a protocol to determine which patients with COVID-19 also have what’s called “cytokine storm syndrome” — a process by which the body’s immune system rapidly releases too many cytokines into the blood.

“The immune system kind of goes awry. It doesn’t act normally. The immune system gets super ramped up, and instead of attacking the virus, it attacks the patient’s own vital organs,” Yadegar explained. “It’s actually [the patient’s] own immune system that’s causing the problem, not necessarily the virus.

“Don’t get me wrong. This is a deadly virus. Just like the influenza virus, it can definitely cause pneumonia. It can definitely cause respiratory failure. If the patient has emphysema or heart failure, it can definitely exacerbate those,” he added. “But this [coronavirus] was doing something unique. This was doing something that I really hadn’t seen much in my 20 years, where it was activating the immune system. And then the immune system was causing all the destruction in the lungs.”

Yadegar noted that, while a virus triggering an autoimmune disease is not necessarily an “unknown thing,” the COVID-19 virus “does it at an extraordinary pace” and to a significant number of patients. He stressed the importance of recognizing that not every patient with COVID-19 will develop an autoimmune disease and that every case must be treated individually.

“One thing that I can’t stress any harder to you and your listeners. There isn’t necessarily one test, and there isn’t one particular treatment plan. Every patient has their own kind of individual disease,” he said. “You can’t treat everyone with the same treatments. There isn’t a one-size-fits-all for this disease. You have to do your due diligence. You have to look at the patient in front of you and then, you know, come up with a treatment for the disease that that patient is manifesting. You can’t just go through the ICU, and start handing out these medicines. If you give this medicine to someone who doesn’t need it, you will surely kill them.”

Watch the video below for more details:

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To enjoy more of Glenn’s masterful storytelling, thought-provoking analysis and uncanny ability to make sense of the chaos,

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Popular heartburn medicine being studied as treatment for coronavirus

Over the past few weeks researchers have been discreetly studying a new potential treatment for COVID-19 — and it might not be what you expect.

The treatment in question is called famotidine, and it’s the active ingredient in Pepcid, an over-the-counter medication commonly used to alleviate heartburn.

Since March 13, researchers at Northwell Health, a network of hospitals in New York, have been enrolling patients hospitalized with COVID-19 into their study of famotidine, which is being delivered through an IV in megadoses nine times greater than the typical over-the-counter dose. The drug is being given in combination with the much-touted antimalarial hydroxychloroquine.

Researchers said some data on safety will be available “in a few weeks,” but did not say when data will be available showing whether the drug combination is effective.

Dr. Kevin Tracey, CEO and president of the Feinsteins Institute for Medical Research at Northwell Health, says the study was being conducted under the radar to avoid media attention as well as a potential depletion of national supply, limiting Northwell’s access to the drug.

More than 180 patients have enrolled in the Northwell study so far. They’ve been given either a two-drug combination of hydroxychloroquine and famotidine, or hydroxychloroquine alone. According to Tracey, hydroxychloroquine was added into the mix because of the drug’s promise back in mid-March — before data emerged about its potential risks.

Tracey said the idea to try famotidine came from a friend and colleague, Dr. Michael Callahan, who had recently visited China and was working with Chinese physicians on a still-unpublished study reportedly showing that the drug benefited patients with COVID-19.

The merit of trying famotidine was supported by additional research, part of a private-public partnership with the federal government, that used the genetic makeup of the virus to pinpoint potentially promising drugs, Tracey said.

The hope is that famotidine will act as a decoy for the virus, so that while the virus is preoccupied with famotidine, it is unable to reproduce itself and spread throughout the body.

Dr. Stuart Ray, professor of medicine in infectious diseases and vice chair of medicine for data integrity and analytics at Johns Hopkins, said he was surprised to hear that researchers at Northwell are studying famotidine in people with COVID-19, and that he is skeptical about preliminary data from China because it has not been vetted in the typical review process.

However, he said he is glad there is some scientific rationale behind the drug. He adds that despite the megadoses, the drug is likely to be safe.

“I think this sort of off-label repurposing is sensible with drugs for which we have a long safety record,” said Ray. “And

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Can A Doctor Assistant Prescribe Treatment?

We are devoted to exceptional pupil schooling, offering thoughtful and inspirational pathways leading to Doctor of Medicine and Physician of Philosophy in Fundamental Biomedical Sciences levels. It is studied individually in temperate climates where these ailments are fairly unfamiliar to medical practitioners and their local medical wants. Many of those lymph vessels run just under the pores and skin and proponents of dry brushing claim that brushing the pores and skin commonly helps stimulate the conventional lymph circulation within the body and assist the body detoxify itself naturally.

One of many 4 founding physicians of The Johns Hopkins Hospital and college of medicine, he created the first medical residency program in the nation, built an environment for selling basic and translational analysis and superior undergraduate medical education by bringing medical students from the classroom to the affected person’s bedside.

For the reason that aim of Orthodox Medicine is simply to take away symptoms of illness somewhat than attain a situation of optimum well being, the elimination of symptoms becomes an end in itself moderately than a means of identifying and correcting the underlying explanation for illness.

Since this medicine labored for cancer, do you think it will also work for a illness called CMT? You’ll not get this relaxation by watching the TV. On a regular basis your body and thoughts want a while off to chill out. This growth within the well being care trade took its toll on the repute of natural cures that are now regarded with speculation, notably on their effectiveness in the treatment of illnesses.

With the Class of 2020, the college’s applied a brand new medical training curriculum that better displays the fashionable U.S. healthcare surroundings and supply models, readying students to follow medicine in a workforce-based, interdisciplinary setting.

Orthodox Medicine treats the physique (person) in isolated elements and believes it has the ability and information to fix an innate (natural) system by interfering in its regular homeostasis (entire body stability) utilizing highly effective man-made chemicals.…

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Poisonous, Herbal Treatment

The formal time period for painkillers is analgesics, a phrase derived from the Greek phrases an (with out) and algia (ache). Muncie is the Faculty’s only campus that is positioned on hospital property, giving medical college students a front-row four-12 months medical education with all the amenities that come with being situated close to the campus of Ball State University, a bustling school setting.

Good day pals this is dr.sukla,we are utilizing dr nagesh medicines from two years to my sufferers, it is best for most cancers on this world. Medical college students must complete a well being questionnaire and bear a medical examination. For further information about learning Medicine at the College of Cambridge see the College of Scientific Medicine web site.

Sepia – Pores and skin yellow, like jaundice; chapping of the skin, or cracks might lengthen deeply into the tissues, and this is aggravated by washing in water; corns with jerking or darting pains; exanthema usually, significantly when it’s disposed to crack; dry itch, often discovering dangerous results where the itch has been suppressed by Mercur.

Visit for medical information and well being information headlines posted all through the day, every single day. Im all for pure remedies myself but I also work with my doctor too. Faculty of Scientific Medicine – The website for the Faculty of Medical Medicine.

The Medical College at Oxford is comparatively small, allowing college students and workers to get to know each other and benefit from a relaxed and pleasant atmosphere. Rural experience: our Faculty of Rural Well being offers medical placements all through central, northern and western NSW.

After examination for indicators and interviewing for signs , the physician may order medical tests (e.g. blood tests ), take a biopsy , or prescribe pharmaceutical drugs or different therapies. At the finish of the day you are trying to steer your interviewers that you’re appropriate for their medical school.…

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Causes, Signs, Treatment, Medication

Many men believe erectile dysfunction means they can’t get it up in any respect, however for some men, the reality is a bit more advanced. 7. After getting out of the shower, pat dry pores and skin and apply a pure fruit oil like rose hip or coconut oil. A healthy weight-reduction plan is a much better resolution that invasive surgical procedure or drugs; eat wise, hydrate and train recurrently to be in one of the best health of your life.

Sign up for a free Medical News At present account to customise your medical and health information experiences. Eating proper meals with correct amount of energy, proteins and vitamins at the right time is very important in preserving mind and body healthy.

Learning to care for a penis builds sexual self-confidence and will increase penis health, so that when it comes time to use that manhood it lives as much as all expectations. A pure residence remedy towards rhinitis of any nature- put four Table spoons of crushed dry onion peels right into a pot of boiling water and inhale over the steam for three-5 minutes.

That is vitally important in a penis health crème, because the nerve endings of a healthy penis are what give a person so much pleasure from sexual activity. If solely we tap into the wealth of health benefits our pure meals supply we would be far much less depending on medicines that deal with one thing and provides rise to another unintended effects.

Physical examination is the initial step in diagnosing a affected person where a health professional acquires pertinent information that results in suspicion of an emanating cancer within the throat. four. Pineapple helps in maintaining a healthy gum. Carvacrol in oregano oil protects in opposition to DNA injury to white blood cells.

No particular person making use of for health protection by means of the individual market shall be discouraged from applying for benefits, turned down for coverage or charged more premium because of health standing, medical condition, mental sickness claims expertise, medical historical past, genetic info or health disability.…

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Penile Rash Treatment

History has it that various medicine goes back 5000 years to Chinese conventional medicine, Indian (Ayuryedic medicine) and similar therapeutic traditions in lots of cultures. Waiting to see somebody that solely administers non-traditional medicine as a substitute of their regular physician will create a delay on this therapy. The remedy used to deal with chest ache in people who have heart disease. In the case of antibiotic therapy, missed doses of medicines aren’t solely the issue of individual patients however are a world risk, because it leads to subtherapeutic publicity of these medicines.

The buyer ought to know the secure use of OTC medicines if they’re administered without medical recommendation. This IU College of Medicine campus offers on-campus housing to med college students. The aim of my weblog is not to promote or promote the products, but to teach and inform these most cancers sufferers what different different remedies available to them and things they should think about earlier than going for chemo.

In fact, some people still sought remedies from local shaman who lacked formal training within the medical occupation. Throughout the enormous field of medicine this exhibition makes an attempt to focus on only some subjects: the physique, genomes, obesity and residing with medical science.

We know that in a pure strategy to heal illness, reminiscent of utilizing herbs and pure ingredients will be eliminate harmful from the physique. A healthcare scientist is involved with the delivery of analysis, remedy, care and help of sufferers in systems of healthcare, versus people in academic research.

Sepia – Skin yellow, like jaundice; chapping of the pores and skin, or cracks may prolong deeply into the tissues, and this is aggravated by washing in water; corns with jerking or darting pains; exanthema typically, significantly when it is disposed to crack; dry itch, typically finding unhealthy effects the place the itch has been suppressed by Mercur.

The FDA-mandated double blind, placebo-controlled medical trials conducted to substantiate the effectiveness of narcotics within the remedy of various medical circumstances have turn into the worldwide commonplace of efficacy amongst healthcare professionals in the final 50 years or so. Furthermore, the ability of narcotics to immediately remediate a illness solely promoted the demand for these treatments amongst customers.…

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