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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

Hartford HealthCare and Connecticut Children’s broaden partnership extending pediatric care

The top executives of Hartford HealthCare and Connecticut Children’s on Monday signed an agreement broadening a 25-year partnership to combine pediatric services with the hospital system’s extensive network across Connecticut.

“This is a continuation and expansion and formalization of an existing relationship,” James Shmerling, president of Connecticut Children’s, said at an announcement outside Hartford Hospital.

Jeffrey Flaks, president of Hartford HealthCare, promised investments and modernization of services at neonatal intensive care units as Connecticut Children’s services are moved into Hartford HealthCare facilities.

“We’re going to work perfect the hand-off when children become adolescents and adolescents become adults for their health care,” he said.

Hartford HealthCare includes two tertiary-level teaching hospitals, an acute-care community teaching hospital, an acute-care hospital and trauma center, three community hospitals, a behavioral health network and a multispecialty physician group.

Stephen Singer can be reached at ssinger@courant.com

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©2020 The Hartford Courant (Hartford, Conn.)

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Pediatric Fractures Shift During Pandemic

Editor’s note: Find the latest COVID-19 news and guidance in Medscape’s Coronavirus Resource Center.

Pediatric fractures dropped by 2.5-fold during the early months of the COVID-19 pandemic, but more breaks happened at home and on bicycles, and younger kids were more affected, new research indicates.

The study of 1745 patients also found that those with distal radius torus fractures were more likely to receive a Velcro splint during the pandemic. Experts said this key trend points toward widespread shifts to streamline treatment, which should persist after the pandemic.

“We expected to see a drop in fracture volume, but what was a bit unexpected was the proportional rise in at-home injuries, which we weren’t immediately aware of,” said senior author Apurva Shah, MD, MBA, of Children’s Hospital of Philadelphia (CHOP) and the University of Pennsylvania Perelman School of Medicine in Philadelphia.

“As time went on, it became more apparent that trampoline and bicycle injuries were on the rise, but at the beginning of the pandemic, we didn’t intuitively expect that,” he added.

“Whenever there’s a major shift in how the world is working, we want to understand how that impacts child safety,” Shah told Medscape Medical News. “The message to get out to parents is that it’s obviously difficult to supervise kids while working from home” during the pandemic, “and that supervision obviously is not always working as well as intended.”

Joshua T. Bram, a medical student, presented the study at the virtual American Academy of Pediatrics (AAP) 2020 National Conference.

Bram, Shah, and colleagues compared patients with acute fractures who presented at CHOP between March and April 2020 with those who presented during the same months in 2018 and 2019.

Overall, the number of patients with pediatric fractures who presented to CHOP fell to an average of just under 10 per day, compared with more than 22 per day in prior years (P < .001). In addition, the age of the patients fell from an average of 9.4 years to 7.5 years (P < .001), with fewer adolescents affected in 2020.

“I think when you cancel a 14-year-old’s baseball season” because of the pandemic, “unfortunately, that lost outdoor time might be substituted with time on a screen,” he explained. “But canceling a 6-year-old’s soccer season might mean substituting that with more time outside on bikes or on a trampoline.”

As noted, because of the pandemic, a higher proportion of pediatric fractures occurred at home (57.8% vs 32.5%; P < .001) or on bicycles (18.3% vs 8.2%; P < .001), but there were fewer organized sports–related (7.2% vs 26.0%; P < .001) or playground-related injuries (5.2% vs 9.0%; P < .001).

In the study period this year, the researchers saw no increase in the amount of time between injury and presentation. However, data suggest that in more recent months, “kids are presenting with fractures late, with sometimes great consequences,” Shah said.

“What has changed is that a lot of adults have lost their jobs, and as a consequence,

Basic Or Pediatric Dentistry

Poor dental health can always have an effect on your self confidence. Many Invisalign certified Orthodontists and Dentists can help progress the therapy and improve your smile with the assistance of the invisalign Expertise. In such instances, the gums where the impacted wisdom teeth are might turn into infected and swollen.

On high of all of this, our healthcare systme is not falling apart. The rationing of healthcare is changing into a difficulty as new applied sciences make it attainable to maintain people alive at great price but with such a poor quality of life that lots of them need to die.

One of the vital efficient treatments for yellow teeth that your dentist will suggest to you is using bleaching trays. Whether or not they’re in personal practice, or work in a salaried place, such as at a hospital, most dentists work full time. Except for having a chunk of tooth left within the gum after an extraction a couple of years in the past, I’ve by no means had a bad experience by the hands of a dentist.

If the pain is from decay on a tooth that you can get to, or if there’s a chip that is inflicting ache, you’ll be able to strive rinsing your mouth with heat salt water, and applying some clove oil to the realm of ache. A affected person requiring a PFM crown needs to have their broken tooth floor down by a dentist to remove the damaged tissue and to create sufficient room to suit the steel and porcelain crown into.

The individuals which are towards Common Health Care are; for one, the docs, due to concern of getting paid much less; republicans, because they don’t wish to share the fee with everybody; the insurance corporations, for fear of getting paid much less.…

Normal Or Pediatric Dentistry

Are you afraid of the dentist? Now, I’m ready of not being able to afford dental care in any respect; even a cleaning is more than I pays. Still, many opponents of common healthcare in America will not be convinced. Many dentists feel that they seem too “salesy” if the workplace calls to comply with up and schedule therapy.

– Typically tumors, most cancers, or trauma may cause the emergency removal of tooth and typically insurance does not cover full restore or implants because they may think about it cosmetic. As soon as a tooth rotted away, these strands of pulp would be seen protruding from the gums.

When individuals snore a lot they go away their mouth open and air dries out the gums, causing them to change into infected. Nevertheless, patients often opt for cosmetic dentistry to enhance enamel whiteness. Dental hygiene practices normally handle to maintain your enamel wholesome.

You can find a veterinary dentist near you by visiting the American Veterinary Dental College web site. In most cases, dental sufferers go to the clinic when in a number of ache and it is subsequently essential that they are attended in the most skilled method and within the shortest period of time potential.

However, in case your gums present a distinct aspect or start to bleed often, it is very important go to a dentist. She or he is within the enterprise of maintaining and fixing teeth-that’s the place the true money is-and so all promotions should replicate the dentist’s companies, not facet jobs.…