April 20, 2019

Your name, title and department. How long have you worked here?
MaJomela ‘Joie’ Nagal, MBA, BSN, RN, CPN, Education Coordinator on 10 WT Neurology/Neurosurgery/Epilepsy Monitoring Unit. I have worked at Texas Children’s Hospital for 11 years.

Tell us how you found out you won a super star award.
I thought I was meeting with Sheranda Fesler, our assistant director of Clinical Practice, Nursing to discuss my FY 2019 goals. She came to meet with me but asked me to go with her to attend to a more urgent matter in a haste so I knew it had to be important. I was surprised to see my colleagues, 10 WT Leadership Team, Emily Weber, Jennifer Sanders and Jackie Ward in the conference room. Sheranda announced that I won Super Star Award. I still could not believe it, I kept looking around the room and I thought for a second that I recognized my husband in the corner. That was nice of Sheranda to invite my husband to come over and be part of my celebration. I had no idea how he was invited but Sheranda explained how she orchestrated the event. I could not explain the overwhelming joy and the mixed emotions but I knew in my heart that I was overjoyed.

What does it mean to be recognized for the hard work you do? How has the organization helped you achieve your personal and professional goals?
I am so honored to be recognized and so grateful to have received this award. It gives me a great sense of confidence to know that my hard work is being recognized. It also is comforting to know that my colleagues appreciate my efforts. It is an honor and a privilege to work here at Texas Children’s Hospital and be surrounded with incredible people to work with. Texas Children’s has a lot of wonderful opportunities to offer and these kept me motivated and continued to inspire me to learn more, improve and grow in my nursing profession. My journey here at Texas Children’s started when I was hired as a staff nurse. Texas Children’s has provided an environment in which front line staff like myself was able to engage and join the unit based and house-wide committees which supported nursing practice and fostered participatory decision making, professional accountability and career growth. I have taken on various expanded roles in the unit level and house wide. After fulfilling several unit-based expanded roles and becoming involved with various projects, I began to see the need and the value as an informal leader. Along the way, I have met amazing people and inspirational leaders who were instrumental in achieving my personal and professional goals. I felt that I could best contribute to advancing the growing and evolving nursing profession through nursing education.

What do you think makes someone at Texas Children’s a super star?
A Texas Children’s Super Star must uphold and exhibit a passion for Texas Children’s mission, must provide quality and excellent clinical patient care, a role model in exhibiting integrity and ethical behavior, and value others by showing respect, compassion and empathy. A super star is someone who is able to take action and initiative to solve problems, someone who is not only willing to embrace change but be the change agent.

What is your motivation for going above and beyond every day at work?
My biggest motivation for going above and beyond is knowing that I work with an amazing team whom I really care about and inspire me to do better each day. It gives me sense of great satisfaction knowing that staff are empowered, equipped with knowledge and training to safely care for the patients and ultimately provide quality and excellent nursing care. It also gives me the sense of fulfillment to witness the journey of a novice nurse, to be there to nurture them on their professional development and until they become experts.

What is the best thing about working at Texas Children’s?
The best thing about working at Texas Children’s is working alongside extraordinary people that I share the same passion serving pediatric population. Knowing that I am part of an organization that is family centered care, full of opportunities, supports nursing practice, advancing in technology and serves not only the children but as well as women in the global community and more importantly leading in patient care, education and research.

What does it mean to you that everyone at Texas Children’s is considered a leader? What is your leadership definition?
I believed that commitment to excellence starts with the leader, so know in your heart that where you are is where you were placed to serve and share your skills and talents. A leader is able to develop others by highlighting their strengths, minimizes their weaknesses and builds upon that. A leader is someone who is an effective communicator, can connect with staff, can foster a positive work culture environment, able to incorporate core measures in daily practice. Someone who is a positive influencer, can lead by example, is reliable and trustworthy.

Anything else you want to share?
I would like to thank my fellow Educator, Heather Morand-Reid who nominated me and to the 10 WT staff and the Leadership Team whom she collaborated with to make this possible. Thank you team!

April 2, 2019

On March 25, the first patient was seen in the new expansion of Texas Children’s Hospital West Campus Sleep Center. Going from six beds on the fourth floor, to nine beds with six available for future expansion on the second floor, the center will now be able to improve access for patients waiting to be treated for sleep conditions.

“We get about 40 to 50 referrals for a sleep study per day,” Neurophysiology Manager Wes Moulden said. “We were looking at anywhere between three to seven month wait times. Now that we have expanded the sleep lab we’ll be able to start diagnosing more children with sleep disorders, and doing sleep studies more efficiently.”

Texas Children’s Sleep Center is one of the few accredited centers in the country specializing in children’s sleep disorders. A sleep disorder is a disruption in a child’s normal sleeping pattern. The sleep center evaluates and treats common sleep disorders in children including problems falling asleep or staying asleep, sleepwalking and abnormal movements during sleep.

“An accredited pediatric sleep center is very rare and all three of our sleep labs have that recognition, Moulden said. “Another unique thing about our sleep center from a diagnostic standpoint, is that all of our technologists are registered technologists who have been formally educated and trained in this skill which is not normally the case.”

Our prestigious credentials also draws the attention of patients from all over the country to our sleep centers. In 2013, Maria Wilson moved her family to Houston from Idaho so that her daughter Sophia could be treated at our sleep center for what was eventually diagnosed as narcolepsy.

“I knew it was going to be a better situation,” Wilson said. “It was very nerve racking and I was nervous, but I knew it was a better move for us to be seeing doctors that have more experience with my daughter’s condition.”

Since being diagnosed and treated frequently at our West Campus Sleep Center for the past six years, Maria says that Sophia is doing much better and is also receiving a great deal of help as they prepare to transition out of the pediatric care system.

“We love Texas Children’s Hospital! After we got established as patients, we got to know the doctors and built relationships with them. That really helped her quality of life when it comes to going to school and functioning better overall,” Wilson said. “Now she’s transferring slowly to adult doctors, and I’m kind of sad. But for us, we love Texas Children’s, we love the doctors and nurses here, and the care that she’s received, so it is comforting to know that we have them in our corner.”

Last week the sleep center team members, administration, and executives gathered on the second floor of West Campus for a ribbon cutting that marked the opening of four sleep clinics, and nine sleep lab beds.

“I wanted to thank all physician leaders, operations leadership, and anyone who had a hand in this much needed expansion,” Texas Children’s Hospital West Campus Vice-President Ivett Shah said. “Texas Children’s is dedicated to improving access to care and this expansion is another way that West Campus continues to provide high-quality care to the West Houston community and throughout the state of Texas.”

The rooms in the sleep center also have the capacity to perform electroencephalogram studies during the day (EEG), in-room monitoring devices, and a host of other quality equipment to ensure sleep studies are carried out properly, along with a scorer/nurse workroom.

“This space was specifically created with the comfort of the patient in mind,” West Campus Respiratory Care and Clinical Support Services Assistant Director Gbolahan Harris said. “Having these clinics and labs in one space helps to reduce patient and family anxiety while normalizing the experience for a sleep study within the sleep center.”

Rooms on the floor also have the flexibility to be converted back into Acute Care beds if West Campus reaches a point where there is a need.

In addition to larger rooms and more workspace, the idea was to increase the number of providers as well. Prior to opening the expansion Lacie Petitto began as the first full-time nurse-practitioner that will be working in the clinic.

“My background is in sleep medicine so I am so excited for the expansion and my new opportunity to treat children in the sleep center,” Petitto said. “When I was approached by the sleep division for this leadership position I was overjoyed to be a part of this opening. This type of work helps feed my passion for sleep medicine and treating patients with sleep disorders.”

Although they are not board certified to read sleep studies, however clinically, Advance Practice Nurses (APN) can see the patients, assess, diagnose, and treat them as well. This allows clinic volumes to open and more patients are seen and treated in a much more efficient way.

“That’s never been done before at Texas Children’s as far as having a full-time nurse practitioner for sleep,” Moulden said. “The center is multi-faceted we have a variety of providers. Some are neurologists, some are pulmonologists, and we are the primary pediatric partner for the Baylor College of Medicine Sleep Fellowship program where we train physicians as well.”

The ultimate goal of the sleep center moving forward is to continue to provide the same high-level services, but on a larger scale.

“Sleep providers should have dreams, and their dreams should come true as they have today,” Texas Children’s Sleep Center Medical Director Dr. Daniel Glaze said. “We anticipate a lot of work, but it will also be a lot of fun. We look for this to be a model for other sleep institutions, and are committed to providing the best diagnostic and overall care for children with sleep complications.”

October 23, 2018

The New England Journal of Medicine (NEJM), one of the nation’s most respected peer-reviewed medical journals, has released a new study highlighting the groundbreaking work being done by the Undiagnosed Diseases Network (UDN), an National Institutes of Health-funded research consortium that includes the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s Hospital, Baylor College of Medicine, Stanford University and other institutions.

Identifying the genes responsible for rare or unknown disorders using traditional approaches is time-consuming work and can take years, or sometimes even decades. And though genetic sequencing is now a routine part of the care people receive for these types of disorders, many patients are still left without a diagnosis. According to the NEJM study, the collaborative UDN model may already be changing that paradigm.

The study reports that the UDN defined 31 entirely new syndromes, and of 382 completed evaluations, 132 patients received diagnoses, a rate of 35 percent – all in just 20 months. The study also found that of the new diagnoses, 37 percent led to non-therapeutic changes in care, such as narrowing of diagnostic testing, and an amazing 21 percent led to changes in therapies for patients.

“This is a major accomplishment in genomic medicine and a giant step forward for these patients and their families,” said Dr. Huda Zoghbi, director of the NRI.

The UDN was established in 2014 with the mission of providing answers for the millions of patients and their families affected by mysterious and rare conditions, but who, after years of extensive testing, still hadn’t received a diagnosis.

The NRI and Baylor have been at the forefront of discovery in the UDN since its inception, jointly serving as one of seven original clinical sites, where doctors and health care providers, ranging from neurologists, immunologists, nephrologists, endocrinologists and geneticists, come together to help find the cause of participants’ symptoms. Baylor, one of the UDN’s two original sequencing cores, currently acts as the network’s sole DNA sequencing site. The NRI and Baylor were also selected to serve as the UDN’s first Model Organism Screening Center (MOSC), which was spearheaded by NRI investigators Drs. Hugo Bellen, Shinya Yamamoto and Michael Wangler.

“The UDN recognized that the resources we had in place and our high throughput made us the ideal candidate to serve as a MOSC site,” Bellen said. “Because of our success with model organisms and the appreciation physicians have for our work, there will likely be a proliferation of MOSC sites in the future.”

In-depth fruit fly studies at the MOSC have helped physicians and scientists identify genes responsible for rare and undiagnosed disorders, leading to the diagnosis of some of the UDN’s most difficult cases. This collaborative effort, which also includes the University of Oregon, has already directly influenced how clinicians care for patients – patients like Avery Reilly.

At only a few months old, Avery began showing signs of a neurologic condition. She could not sit until age 3 or crawl until age 5, and today she cannot walk or speak. An appointment with Texas Children’s neurologist Dr. Timothy Lotze revealed Avery had poor muscle tone, which was delaying milestones. That initial appointment led to years of doctor visits and countless tests, all of which failed to reveal the cause of Avery’s developmental and speech delays.

Then in 2014, the Reillys heard about the NRI’s involvement with the UDN and submitted Avery’s case. For the first time in years, the family had hope. Using exome sequencing, researchers discovered Avery had a new type of genetic mutation. Then a team at the MOSC, led by Wangler, studied the mutation in a fruit fly model to see how the genes were affected. At long last, the Reillys had a diagnosis. What’s more, the discovery of Avery’s mutation led to a change in her medications, which could help slow the progression of her disease.

“The fact that we are able to help the UDN accelerate science to find actionable changes in therapies is very exciting,” said Zoghbi. “Helping people, and working to solve severe medical problems through basic research, and through unfettered collaboration, is what our work is all about.”

The NEJM study is the first to provide a detailed description of the inner workings of the UDN. It presents an in-depth analysis of the referral and acceptance patterns, diagnoses, impact rates and follow-up scientific investigations of 1,519 cases that were referred to the UDN in the last two years.

As the study points out, the most unique feature, and perhaps the biggest contributor toward UDN’s success, is its model of multi-institutional collaborations. Teams of researchers and physicians from participating institutions all over the nation leverage their multidisciplinary expertise and resources to quickly find specific diagnoses for patients with extremely challenging clinical cases, with no additional cost to the patients.

In addition to the original clinical sites and sequencing cores, the UDN also included a coordinating center as part of its phase I deployment. In 2015, a web-based portal, the UDN Gateway, was launched for patients and families to participate in UDN. The network recently expanded its footprint from seven to 12 clinical sites and also added a central biorepository, a metabolomics core and a new MOSC site.

Though the UDN’s larger focus is currently gene discovery, it’s the work being done at the NRI and Baylor that will set up the network’s next evolution.

“Once a gene is discovered, it’s natural that the focus should shift to finding out what the genes do, what the molecular mechanisms are, if they’re linked to other diseases, or if they can be manipulated with drugs,” Bellen said. “That’s what we’re doing at the NRI and Baylor now, and that will be instrumental in the next phase for the UDN. When that time comes, we’ll be ahead of the curve.”

Learn more about the recent research breakthroughs and patient success stories from the UDN and MOSC.

About the New England Journal of Medicine
For over 200 years, the New England Journal of Medicine has rigorously vetted and compiled the latest medical research in support of physicians and their patients. From the first uses of anesthesia to the most recent cardiology and cancer treatments, the New England Journal of Medicine has helped generations of clinicians enhance their knowledge and improve patient care.

Today, with rigorously peer-reviewed research, topical reviews, interactive clinical content and cases, the New England Journal of Medicine is the trusted source for essential findings in medicine.

September 10, 2018

Texas Children’s Hospital has been recognized as a Clinical Research Center of Excellence (COE) for CDKL5 Deficiency Disorder and is one of only five pediatric institutions in the country to receive this honor.

The International Foundation for CDKL5 Research selected Texas Children’s and its academic partner Baylor College of Medicine as a Center of Excellence for its medical expertise and extensive clinical research experience in the field of Rett syndrome and Rett-related disorders including CDKL5 Deficiency Disorder.

“It’s an honor for our hospital to be chosen as a center of excellence for CDKL5 deficiency disorder research,” said Dr. Bernhard Suter, who is the director of the CDKL5 Center of Excellence at Texas Children’s. “We look forward to collaborating with other centers across the country to accelerate CDKL5 research in order to provide the best care possible for these patients.”

Once considered an atypical form of Rett syndrome due to its overlapping symptoms and similar medical needs among patients with either condition, the symptoms associated with CDKL5 deficiency disorder and its genetic cause are distinct from those of Rett syndrome. CDKL5 deficiency disorder is now considered a separate condition.

The CDKL5 gene provides instructions for making a protein that is essential for normal brain development and function. When the CDKL5 gene is mutated, patients with CDKL5 deficiency experience a variety of symptoms including difficult-to-control seizures, repetitive hand movements and severe neuro-developmental impairment. Seizures usually begin within the first three months of life, and can appear as early as the first week after birth.

As a Center of Excellence, Texas Children’s multidisciplinary CDKL5 clinic (housed in our Rett Syndrome Clinic) provides comprehensive care to patients by allowing them to see several specialists in the same clinic visit – including neurology, genetics, GI, pulmonology, physical medicine and rehabilitation – resulting in more personalized treatment plans for newly diagnosed patients.

“After an extensive search of top notch medical facilities, we found that Texas Children’s Hospital was the perfect pairing of medical expertise and research initiatives into this condition that so tragically affects those suffering with CDKL5,” said IFCR President Karen Utley, who also is a parent of a child with CDKL5. “We are proud to partner with Texas Children’s Hospital in order to improve the quality of life and to find a cure for our children.”

July 17, 2018
On July 9, Texas Children’s Hospital and the Angelman Syndrome Foundation celebrated the official opening of the Angelman Syndrome Clinic at Texas Children’s, one of only seven Angelman syndrome-specific clinics in the United States – and the first in Texas.

Several patient families gathered on the 16th floor of Wallace Tower to attend the ribbon cutting ceremony. Debbie Sukin, the daughter of the late Dr. Ralph Feigin, delivered the opening remarks and shared how this new clinic will benefit her 16-year-old son Jacob and so many other children diagnosed with Angelman Syndrome.

“The hardest part of advocating and managing a child with Angelman Syndrome is trying to understand what might be wrong when they aren’t feeling right, looking right or acting right,” Sukin said. “We’re confident that this multidisciplinary clinic here at Texas Children’s will provide all children the expertise and best chance at meeting their maximum potential and provide guidance to ensure the best outcome.”

Occurring in one in 15,000 live births, Angelman syndrome is a neurogenetic disorder often misdiagnosed as autism or cerebral palsy that causes severe neurological impairment. Often times, these patients experience developmental delays, lack of speech, seizures, and walking and balancing disorders.

With the creation of this new clinic established through the generosity of donors and in partnership with the ASF, patients and their families now can access multiple specialists – experts in Angelman syndrome, clinical geneticists, neurologist, psychiatrist, speech language pathologist, genetic counselors and social work – in one setting rather than visiting multiple locations across the nation to manage and treat this disease.

“Thanks to the help of the ASF, we are proud to further our mission of providing very specialized care for patients with Angelman syndrome,” said Chief of Neurology Dr. Gary Clark. “Given the science that’s being developed at the NRI at Texas Children’s in genetics and neurology, we are making great strides in helping these patients.”

The ribbon cutting event also included remarks from Dr. Carlos Bacino, chief of the Angelman Syndrome Clinic, Carrie Rys, assistant vice president, and Eileen Braun, executive director of the Angelman Syndrome Foundation, whose organization donated $50,000 to advance Angelman syndrome research at Texas Children’s.

“Opening this clinic brings us one step closer to our commitment to establish a network of clinics across the country,” Braun said. “We could not be more proud to support Texas Children’s and their remarkable team of professionals in bringing this clinic to life. We look forward to impacting the lives of so many more families.”

Click here to learn more about our Angelman Syndrome Clinic at Texas Children’s.

June 26, 2018
Texas Children’s Hospital has once again been named as a national leader among pediatric institutions by U.S. News & World Report in their recently published 2018-19 edition of Best Children’s Hospitals.

Ranked fourth among all children’s hospitals nationally and one of only 10 hospitals to achieve the Honor Roll designation for the tenth straight year, Texas Children’s is the only hospital in Texas – and the entire Southern region of the U.S. – awarded this coveted distinction.

“Each year, our Texas Children’s team exhibits incredible strength and kindness, as well as passion, caring for the inspirational children and families we serve,” said Texas Children’s President and CEO Mark Wallace. “I believe this is one reason why we continue to maintain the respect and reputation as one of the best hospitals in the nation, and the destination for pediatric care in Texas.”

In addition to ranking children’s hospitals overall, U.S. News & World Report also ranks the top 50 pediatric hospitals in 10 major sub-specialty areas. To be considered for the honor roll distinction, a hospital must have high rankings in at least three sub-specialties. For the second straight year, Texas Children’s Heart Center ranks No. 1 in the nation for pediatric cardiology and heart surgery. Texas Children’s Pulmonology ranks as the best program in the country for children with lung diseases.

Texas Children’s has 8 subspecialties ranked in the top 10, and the hospital improved outcomes across all sub-specialties. There are approximately 190 children’s hospitals in the U.S. and this year, 86 of the 189 surveyed hospitals were ranked among the top 50 in at least one sub-specialty. The 2018-19 Best Children’s Hospitals Honor Roll recognizes the 10 hospitals with the highest rankings across all sub-specialties. Here are a few highlights of this year’s rankings for Texas Children’s:

  • Cardiology and Congenital Heart Surgery is again no. 1 in the nation and received the top score in externally reported risk-adjusted operative mortality for congenital heart surgery.
  • Pulmonology, which first debuted in the top spot in the 2016 rankings, is now again ranked no. 1 in the nation. We received the top score in several asthma outcomes and structure metrics, such as mean LOS for asthma patients.
  • Neurology and Neurosurgery moved from no. 4 to no. 3, receiving the top score in several outcomes metrics, such as 30-day readmissions for craniotomy and Chiari decompression and complication rate for epilepsy surgical procedures.
  • Nephrology also moved from no. 4 to no. 3, with the top score in one-year kidney transplant graft survival and hemodialysis catheter-associated bloodstream infections.
  • Urology moved from no. 6 to no. 4, propelled by the top score in unplanned hospital admission for urologic issues within 30 days of surgery, as well as significant improvements in hypospadias and revision surgeries.

Texas Children’s, working closely with our academic partner Baylor College of Medicine, continues to pioneer advancements in pediatric health care and earns the U.S. News honor roll distinction by being ranked among America’s best in:

  • #1 Cardiology and Congenital Heart Surgery
  • #1 Pulmonology
  • #3 Neurology and Neurosurgery
  • #3 Nephrology
  • #4 Gastroenterology and GI surgery
  • #4 Urology
  • #6 Cancer
  • #6 Diabetes and Endocrinology
  • #15 Orthopedics
  • #21 Neonatology

This year’s rankings are the results of a methodology that weighs a combination of outcome and care-related measures such as nursing care, advanced technology, credentialing, outcomes, best practices, infection prevention and reputation, among others.

“From a measurement perspective, our survey results demonstrate how hard we’re working as an organization to deliver high quality care to our patients,” Wallace said. “The more consistently we deliver high quality care and the safer we deliver that care to our patients, the better their outcomes are, and the better our overall numbers are.”

Our results continue to reflect the diligent efforts of a solid structure focused on the U.S. News survey. The process of compiling and refining our data is an ongoing challenge, which will continue to improve under the excellent leadership of Trudy Leidich, Elizabeth Pham and the entire USNWR team.

The 2018-19 edition of Best Children’s Hospitals is available online at www.usnews.com/childrenshospitals.

June 12, 2018
As Texas Children’s continues to expand its physical footprint as a leader in pediatric care, Texas Children’s Neurology Program is growing by leaps and bounds. As one of the largest pediatric neurology service providers in the nation, more than 30,000 patient encounters occur each year at Texas Children’s Neuroscience Center.

Ranked No. 4 nationally in neurology and neurosurgery by U.S. News & World Report, patients are treated for a number of neurological conditions in our 13 specialty clinics including epilepsy, cerebral palsy, Rett syndrome, Batten disease and movement disorders. Many patient families are referred to Texas Children’s for our integrated expertise and multidisciplinary team approach in diagnosing and treating childhood neurological disorders. Recently, the Neurology division expanded its services with the opening of the Angleman Syndrome Clinic to treat children with this very rare, neurogenetic disorder.

“Over the last decade, our neuroscience programs have experienced phenomenal growth,” said Chief of Neurology and Developmental Neuroscience Dr. Gary Clark. “In 2004, the division had 5,000 patient encounters per year. Since then, we have more than quadrupled our patient encounters to 31,500 in 2017. More than half of these patients are seen in the community, allowing for greater access to care.”

In 2017, the neurology service received 1,300 new patient referrals each month which is a 30 percent increase from last year. To meet the increased demand for these services and to accommodate the growing patient population, the pediatric neurology division faculty expanded from 9 to 52 neurologists.

While enhancing access to patient care remains a top priority for Texas Children’s, one way the Neurology service is helping to achieve this collaborative milestone is through the implementation of telemedicine, an interactive telecommunications system that uses real-time video technology to link patients with their primary care and specialty providers. Instead of making the trek to the Texas Medical Center, patients can now “see” their neurologist without ever leaving their primary care clinic.

“Through an HRSA grant, we have seen children with epilepsy in one of our medical homes via this new technology and completed more visits than any other grant recipient,” said Brian Cordasco, practice administrator for Neurology. “This successful experiment will serve as a blueprint for future growth of telemedicine at Texas Children’s. Six faculty offices are now equipped with telemedicine capabilities.”

To balance outpatient services with the increasing demand for inpatient care, the Neurology Service has also developed an inpatient service rotation for physicians at Texas Children’s Hospital West Campus. Just like Texas Children’s Medical Center Campus and Texas Children’s Hospital The Woodlands, neurologists and epileptologists take turns working out of clinic to take neurology consults at the hospital.

Partnerships advance clinical research in neurology

The neurology division’s partnership with the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s under the leadership of Dr. Huda Zoghbi also has helped to advance clinical and basic science research leading to the development of new treatments for neuro and developmental disabilities.

Nearly $185 million has been raised to build and partially endow the NRI, and the hospital’s long-time philanthropic partner, The Blue Bird Circle, has raised funds (initial gift $1M for four years) to form and partially endow the Blue Bird Circle Clinical Research Center in which NRI discoveries are realized through clinical trials.

The Blue Bird Circle has been the catalyst for so much of what has happened in pediatric neurology in the past 20 years at Texas Children’s. Clinical research protocols have grown from 9 to 63 over the 8-year existence of the Blue Bird Circle CRC. The Blue Bird Circle has raised in excess of $1.2 million annually to directly fund clinical treatment, training and research in pediatric neurology at Texas Children’s.

Through these collaborative research partnerships, the neurology team has made significant progress:

  • Texas Children’s is one of the few sites in the country that provides therapy to patients with an enzyme deficiency in the form of Batten disease by surgically injecting the synthesized enzyme directly into the brain thereby halting this degenerative form of Batten disease.
  • Texas Children’s treats patients with spinal muscular atrophy, a disease where the spinal cord degenerates due to a defective SMN1 gene. This treatment involves injecting a DNA molecule into the spinal fluid that turns a latent gene into an effective one that makes the deficient protein.

“We are contemplating similar treatments for Angelman and Rhett syndromes,” Clark said. “We are so grateful to Texas Children’s Blue Bird Circle Research Center in collaboration with the Jan and Dan Duncan Neurological Research Institute at Texas Children’s for helping us make these research breakthroughs possible, and I am excited about what the future will hold for pediatric neurology.”