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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Bauer Smiles – Wheaton Orthodontist, Dentist, Pediatric Dentist

Call 630-665-5550

We have you covered! We believe that you and your family will love our practice, as so many of your wonderful Wheaton neighbors already do! Our office offers a complimentary first visit with the dentist, if desired, so you can view our office and meet the dentist or orthodontist to see if we are the right dental and/or orthodontic office for you. You can also Schedule Online right now!

Bauer Dentistry and Orthodontics motto

We are here to serve the dental and orthodontic needs of your entire family for their entire life. Our team believes that exceptional dental care starts with a genuine relationship.
We will listen to your concerns and educate you on your orthodontic and dental options. Our highly-trained doctors and staff are extremely friendly and welcoming, treating you as though you are part of our family.
We are a multi-specialty dental office located in Wheaton, IL, that utilizes the best technology available so that we can treat almost any oral health care concern in the same location. We will often see an entire family at the same time, a real time saver for moms and dads.


Need a Wheaton orthodontist or dentist? We really are a one stop shop for your family’s dental needs!

Wheaton pediatric dentist, Dr. Lynse Briney, sees children of all ages and special needs patients. She uses nitrous gas, can do full sedation dentistry in a hospital setting, and of course has TVs in the ceiling for the kiddos! She does composite fillings, tooth colored dental crowns, and does tooth extractions when needed.

Dr. Danielle Bauer is our Wheaton orthodontist. She provides Invisalign, Invisalign teen, Incognito lingual braces, and uses top of the line self-ligating Damon and Damon clear braces. Since she uses Wilckodontics and AcceleDent, you have great options to have accelerated orthodontics!

Dr. Bryan Bauer, our cosmetic dentist, handles all dental care including root canals, porcelain veneers, and laser dentistry (including venous lake treatment). He also provides teeth in a day, all on 4, implant supported denture, and other more complex dental implants, and of course the tooth extraction itself. What a great choice for your Wheaton dentist!

We are conveniently located on Roosevelt road in Wheaton, IL and have plenty of private parking. Schedule Online

You have found the right place! A great Wheaton pediatric dentist, orthodontist, and Wheaton dentist all together!

Call 630-665-5550 or email us today to start your dental care with a great group of people! Since we offer complimentary visits, you can meet the staff and see our Wheaton dental office in person!

Check out our dentist reviews page or the reviews on facebook, google, and yelp to hear what our current Wheaton patients are saying!

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What is a Pediatric Dentist?

Pediatric dentists are dedicated to the oral health of children from infancy through the teen years. They have the experience and qualifications to care for a child’s teeth, gums, and mouth throughout the various stages of childhood.

Children begin to get their baby teeth during the first 6 months of life. By age 6 or 7 years, they start to lose their first set of teeth, which eventually are replaced by secondary, permanent teeth. Without proper dental care, children face possible oral decay and disease that can cause a lifetime of pain and complications. Today, early childhood dental caries—an infectious disease—is 5 times more common in children than asthma and 7 times more common than hay fever.

What Kind of Training Do Pediatric Dentists Have?

Pediatric dentists have completed at least:

  • Four years of dental school
  • Two additional years of residency training in dentistry for infants, children, teens, and children with special needs

What Types of Treatments Do Pediatric Dentists Provide?

Pediatric dentists provide comprehensive oral health care that includes the following:

  • Infant oral health exams, which include risk assessment for caries in mother and child
  • Preventive dental care including cleaning and fluoride treatments, as well as nutrition and diet recommendations
  • Habit counseling (for example, pacifier use and thumb sucking)
  • Early assessment and treatment for straightening teeth and correcting an improper bite (orthodontics)
  • Repair of tooth cavities or defects
  • Diagnosis of oral conditions associated with diseases such as diabetes, congenital heart defect, asthma, hay fever, and attention deficit/ hyperactivity disorder
  • Management of gum diseases and conditions including ulcers, short frenulae, mucoceles, and pediatric periodontal disease
  • Care for dental injuries (for example, fractured, displaced, or knocked-out teeth)

Where Can I Find A Pediatric Dentist?

Pediatric dentists practice in a variety of locations including private practices, dental schools, and medical centers. Your pediatrician can help you find a pediatric dentist near your home.

Pediatric Dentists — The Best Care For Children

Children are not just small adults. They are not always able to be patient and cooperative during a dental exam. Pediatric dentists know how to examine and treat children in ways that make them comfortable. In addition, pediatric dentists use specially designed equipment in offices that are arranged and decorated with children in mind.

A pediatric dentist offers a wide range of treatment options, as well as expertise and training to care for your child’s teeth, gums, and mouth. When your pediatrician suggests that your child receive a dental exam, you can be assured that a pediatric dentist will provide the best possible care.

How to Find a Pediatric Dentist:

To find a dentist to care for your child visit the
American Academy of Pediatric Dentistry web site or
insurekidsnow.gov. ​

The information contained on this Web site should not be used as a substitute for the medical care and advice of your pediatrician. There may be variations in treatment that your pediatrician may recommend based on individual facts and circumstances.

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Winning Smiles Pediatric Dental Care

We’ve worked hard to create a space that’s welcoming, relaxing, and most of all, fun, for you and your child.  In fact, we like to think of it more as a dental “home” than a dental “office.”  The American Academy of Pediatric Dentistry defines a dental home as an “ongoing relationship between the dentist and the patient, inclusive of all aspects of oral health care delivered in a comprehensive, continuously accessible, coordinated, and family-centered way.”  From the caring and knowledgeable dentist and staff, to the child, parent and eco-friendly equipment and furnishings, we’ve done our best to make you feel like family in a place that will become your child’s dental home for years to come.  Our mission is to provide the kids of Pittsburgh with high quality dental education and treatment needed to grow into happy, healthy adults. 

Take a look around.  Learn more about our unique ability to connect with and provide top notch dental care to children of all ages.  Check out our wide range of services and how we make them as comfortable as possible.  Meet Dr. Erin and browse the gallery to find out how we, along with Artemis Environmental, a local “green” construction and design company, have thoughtfully planned and built (from scratch!) a comfortable, healthy and safe space for you, your kids and our planet. 

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Pediatric Dentistry

Recently, I was asked to speak on marketing and social media.  Our large group pediatric dentistry and orthodontic practice requires a solid marketing plan to keep the practice healthy.  We actually spend more time that I think we should on what is vaguely called “marketing.”

Seth Godin, guru of the marketing world, a while back recommended aspiring writers to “give away” their first book?  Really? I can see writers saying, “No way.  I spent thousands of hours of effort on this thing to just give it away for free”.  How can this work?  As he explains, you gain a following, you gain (deserved) notoriety as an expert and authority.  Then, when your second book comes out, you have a built-in audience.

We as professionals, usually view with disdain trying to “sell” our services.  We want to spend time on dentistry and patient care.  We are above such pandering.  Still, in the modern marketplace, if you do not let people know who you are, what you do, and that you are accepting and want new patients, your practice will slowly whither away.  This is especially true of practices like Pediatrics and Orthodontics.  Your patients eventually grow up and need to be replaced with younger patients to maintain your practice.

I will not go into great detail on dental practice marketing, that would take several books worth of text and seminars to get all that across.  I will say, the number one way to grow your practice is by giving good service and the word of mouth that is generated from that.  Sometimes, however, that is not enough.  It is obvious that part of our profession is to give of our talents.  We do charity work, do overseas mission trips and work for free at local charity clinics.  There is great value just in that.  I think the general public has no real idea how much free service dentists give away every year, sometimes not by choice, -but I will not get into that here.

What I have tried to do with this blog is inform and entertain.  I don’t earn any money off this blog.  In essence, I give it, the content, away for free.  Why?  Well, first off, I just enjoy informing and educating.  I enjoy writing.  Secondly, in a business and marketing sense, reputation leads to increased business, to increased patients wanting your services.  They know you, they respect you because they know you know your stuff.

Other professionals are doing this and vary from musicians to English tailors.  I follow  a music group called Postmodern Jukebox.  They make music videos and post them online.  They are very good, but offer these online for free.  Now, after gaining notoriety, they are offering their songs on iTunes and are starting an American and European tour.  Another musician, Christopher Bill, a trombone player, makes free videos on his websites.  You may have heard of his version of “Happy.”  He now has an album on iTunes and is well known enough I

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Characteristics For Kids Dentist Or Pediatric Dentist

As a couple and parents, you want your child to feel comfortable when you bring him or her to the dentist. This feeling of comforts starts with the waiting room. Normally, kids do not have a similar level of patience that adults do when it comes to waiting for the appointment. Waiting is seemed boring and painful even terrorize if they are already confused about their visit. Hence, a child-friendly dentist clinic should contain toys and other items which is considered as an interesting fact in the child are suitable for any kids. These requirements are not only fulfilled in the dentist clinic, instead, but some requirements are also fulfilled for the dentist when they treat any kids.

Qualities and Characteristics of a kid’s dentist

There are several qualities and characteristics to being a pediatric dentist such as

Love Children – Pure love and concern for kids should be the highly attribute to look for in your kid’s pediatric dentist.

Patient – this is very important for any pediatric dentist that he and she should have a lot of patience goes without saying.

Authoritative – a dentist should be able to provide meaningful instructions that children will be able to follow.

Compassionate – the dentist should be showing compassion with children, it is very useful for any children who visited for their treatment.

Approachable and Kind – A friendly with smiling face and a warm handshake can go a long way. Always looking for those dentists who welcomes new patients with excitement and kindness.

Engaging – considering those dentists is the best who can capture the attention of children and get them to listen to what the child asks them.

Knowledgeable – it means not about dentist education, it is a must for every dentist who is qualified, instead, it means he and she know the kid’s feelings and emotions and understand child nature.

Has a Kid-Friendly office and clinic – the office place is important as the dentist. The office look has become a playful atmosphere such as fun decorations, attractive cartoons playing on the TV and toys should all be part of the pediatric dentist’s clinic.

Advises the children – The dentists who advise the children on how diet and drinks affect teeth and on the right care of teeth are more effective.


Finding a pediatric dentist for your kids is a significant part of helping them develop good oral hygiene habits at a young age. Dr. Najla Najeeb is one of the passionate dentist who has treat many pediatric cases in her professional life. She is very wonderful characteristics about understanding kid’s nature and she loves to treat kids with a friendly environment for better oral satisfaction. 

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Pediatric Dentists in Indianapolis – Drs. Papineau and Farthing

7725 S Emerson Ave
Indianapolis, IN 46237

Phone: 317-882-7694
Fax: 317-882-8234

Welcome to the pediatric dental office of Scott D. Papineau, PharmD, DDS and Annette T. Farthing, DDS. We provide specialized dentistry for children and adolescents in a “child-friendly” environment. As pediatric dentists, we focus on preventive care to help each child grow a healthy smile that will last a lifetime. Serving infants, children and teens in Indianapolis, Greenwood and Columbus, IN.

Drs. Papineau and Farthing have extensive training and experience in treating the dental needs of infants, children and teenagers. Our practice utilizes the most modern and effective methods for routine dental cleanings and restorations as well as dental emergencies and trauma, oral conscious sedation, IV sedation, and treatment under general anesthesia. Additionally, our practice is equipped with the knowledge and compassion to treat children with special developmental or medical needs.

It is our desire that your child’s first visit to the dentist be as positive, relaxed and enjoyable as possible. We want to help you establish a routine of dental care that will last a lifetime. Finding a dental home will help to maintain excellent oral health throughout childhood. We cannot wait to welcome you to our office.

Appointment Request at a Pediatric Dentist in Indianapolis, Greenwood and Columbus, IN 
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Dr. Papineau and Dr. Farthing are pediatric dentists in Indianapolis, Indiana serving infants children and teens in the surrounding

cities of Greenwood and Columbus, IN

7725 South Emerson Ave., Indianapolis, IN 46237 – Phone: 317-882-7694 – Fax: 317-882-8234

Copyright © 2013 Annette T. Farthing, DDS, Scott D. Papineau, PharmD, DDS and
Smile Savvy. All Rights Reserved.
Disclaimer | Privacy Policies | Accessibility

Pediatric Dental Website Designed by
Smile Savvy
(a sister company of Dentists4kids.com)

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Pediatric Dentist in Kissimmee Florida

Our practice offers complete dental care for children in a comfortable and relaxing atmosphere. We treat children of all ages; however, we do not force any child to do anything they refuse. We postpone of necessary, procedures until that child is more comfortable to allow us the opportunity to treat them. That is why Dr. Hernandez believes in the age one Dental home. The majority of early caries does not require anesthetic.

We work on “kids time”, which means that we will not rush, but allow as much time as necessary for the child to be comfortable.

Professional Services Offered:

pediatric dentist

  • White fillings
  • Silver fillings
  • Prophy
  • Flouride Treatments
  • Oral Hygiene Instructions
  • Extractions
  • Sealants
  • Digital X-Rays
  • Pulpotomies
  • Stainless Steel Crowns

We also offer Interceptive Orthodontics: Habit appliances, Corrective appliances, and Space Maintainers

New Smiles Welcome

Dr. Iris Hernandez welcomes your questions and is happy to provide feedback. Our team consistently works to go the extra mile for your children by using the latest technology and materials and enhancing our skills through continuing education.

Our Amenities

  • Se Habla Espanol!
  • Refreshments
  • Television & Movies
  • Handicap Access
  • Free Parking

We Provide Excellent Care for Special Needs Patients

special needs pediatric dentistryAll children are welcome to Kissimmee Childrens Dentistry and deserve quality dental care. Many children find going to the dentist difficult or traumatic. Opposition to basic dental hygiene, limited or poor diet, physical disability, sensory issues, and resistance to professional dental care can be obstacles to oral health that we understand.

Our pediatric dental office serves children with ASD. We fully understand that children with AUTISM sometimes have difficulties at the dentist.  Many children on the spectrum have severe oral aversions and hypersensitivity and we will help your child to feel comfortable during your dental visit.

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Department of Pediatric Dentistry – UW School of Dentistry

Resident dentist with young patient

In 2010, the University of Washington and Seattle Children’s joined forces to combat the growing crisis of childhood dental disease by combining health services, education, research and public policy under one roof at The Center for Pediatric Dentistry (CPD). In 2013, the Department of Pediatric Dentistry relocated to the CPD.

From infant check-ups and early screening to parental education and special needs access, the CPD equips parents with the service and knowledge to ensure a healthy child and uses a multi-disciplined approach to solving the rampant issues of childhood oral health.


The mission of the Department of Pediatric Dentistry is to promote the health of infants, children, adolescents, and persons with special needs, through education, research, and service, by:

  • Educating pediatric dentists for a lifetime of continuous learning and leadership.
  • Generating new knowledge through basic and clinical research.
  • Promoting effective preventive measures to ensure a lifetime of oral health.
  • Collaborating with other disciplines and organizations to develop policies to increase access to oral health care for all people.


Our vision is to be the recognized international leader in pediatric dental education, research, and the development of service delivery models and to be the leader in establishing mechanisms for policy change to dramatically mitigate the unmet need in the management of dental caries in children worldwide.

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Pediatric Dentist Atlanta Georgia Area

Choosing a dentist is a crucial determination. There are lots of compelling reasons to decide on dentistry as a career. In the a hundred Finest Jobs ” record of 2018, Dentist is #2, Orthodontist is #5, Oral and Maxillofacial Surgeon ties for #8, Prosthodontist is #16, Dental Hygienist is #17 and Dental Assistant is #98.

A dentist treats a affected person with the help of a dental assistant. Approximately ninety% of all dentists are engaged in delivery of care by personal practices. four. Root Canal – Root canal is a procedure that’s used by dentists to take away infected tissues within the tooth pulp.

Moreover, the vary of therapies available on the NHS could also be restricted, so that is one thing else to consider once you’re excited about your funds for dental care. Productive Dentist Academy understands what it is prefer to spend a decade in dental school, earn your degree, and be enthusiastic about finally beginning your own practice – only to finish up stressed and drowning in debt.

Dental procedures could cause fear and nervousness in many patients, and the perfect dentist can be alert to those emotions and show compassion earlier than, during, and after a process. To find out your eligibility, you may be supplied with a free oral well being evaluation by a Certified Dental Practitioner in your area.

At Graf Household Dentistry, Dr. Thomas Graf , Dr. Michelle Graf , and our group love serving to individuals develop healthy smiles. The common wage for dentists in 2015 topped $158,310, whereas these with years of experience can anticipate to earn much more. New thrilling scientific breakthroughs in gene remedy and biotechnology offer dentist an opportunity to protect their affected person’s smiles and self esteem.

The remainder may specialize as pediatric dentists (focusing on dentistry for children and particular-needs sufferers); periodontists (treating gums and bone supporting the tooth); prosthodontists (replacing lacking teeth with everlasting fixtures, corresponding to crowns and bridges, or with removable fixtures equivalent to dentures); endodontists (performing root-canal therapy); oral pathologists (diagnosing oral diseases); oral and maxillofacial radiologists (diagnosing illnesses in the head and neck via using imaging applied sciences); or dental public health specialists (selling good dental health and stopping dental ailments inside the group).…

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