About The Practice

Serving Texas Children's Concerns about Neurology, Epilepsy Developmental & Sleep Disorders. Advanced spasticity management.

The Houston Area ( Bellaire Katy Sugar Land Richmond Missouri City Cypress The Woodlands Lake Jackson)

The Greater San Antonio Area ( New Braunfels Seguin Central Texas)

Dr Joshua Rotenberg. Board Certified in Neurology with Special Qualifications in Child Neurology.

Dr. Rotenberg has added subspecialty board certification in epilepsy AND sleep disorders (American Board of Psychiatry & Neurology-Child Neurology).

Member - American Epilepsy Society

Member - American Academy of Cerebral Palsy & Developmental Medicine

Texas Medical & Sleep Specialists - Children & Adults Welcome. WWW.TXMSS.COM 713-464-4107




Thursday, November 20, 2014

Ataxia Trial for Orphan Drug: Cabaletta for the Treatment of Spinocerebellar Ataxia Type 3


Bio Blast Pharma Receives Orphan Drug Designation From U.S. FDA for Cabaletta for the Treatment of Spinocerebellar Ataxia Type 3



GlobeNewswire

TEL AVIV, Nov. 19, 2014 (GLOBE NEWSWIRE) -- Bio Blast Pharma Ltd. (ORPN), a clinical-stage biotechnology company committed to developing clinically meaningful therapies for patients with rare and ultra-rare genetic diseases, announced today that it has been granted Orphan Drug Designation by the U.S. Food & Drug Administration (FDA) for Cabaletta for the treatment of Spinocerebellar Ataxia Type 3 (commonly known as SCA3 and Machado Joseph disease). This is the second indication for which Bio Blast's Cabaletta has received such designation.
Cabaletta is a chemical chaperone that protects against pathological processes in cells. It has been shown to prevent pathological aggregation of proteins within cells in several diseases associated with abnormal cellular-protein aggregation. Cabaletta has demonstrated efficacy in preclinical cells and animal models of SCA3 and other PolyA/PolyQ diseases, including Occulopharyngeal Muscular Dystrophy (OPMD) and Spino bulbar cerebellar ataxia (SBMA, or Kennedy's disease). Bio Blast plans to make clinical progress in each of these indications in 2015.
"This orphan drug designation lays the foundation for what we believe are very favorable conditions to continue to pursue our clinical plans and reflects our commitment to those patients suffering from rare diseases for which there are few or no therapeutic options. It is yet another step on Bio Blast's route to create and capture value from our research and clinical work," stated Dalia Megiddo, MD MBA, Chief Executive Officer of Bio Blast. "We currently are conducting a phase 2 clinical trial to test the efficacy of Cabaletta in SCA3 and plan to start our SCA3 pivotal study in 2015."
About Orphan Drug Designation
Orphan drug designation is granted by the FDA Office of Orphan Products Development (OOPD) to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The designation provides the drug developer with a seven-year period of U.S. marketing exclusivity upon approval of the drug, as well as tax credits for clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance and waives the Prescription Drug User Fee Act (PDUFA) filing fees.
About Bio Blast Pharma
Bio Blast Pharma is a clinical-stage biotechnology company committed to developing clinically meaningful therapies for patients with rare and ultra-rare genetic diseases. Founded in 2012, the company is rapidly building a diverse portfolio of product candidates with the potential to address unmet medical needs for incurable diseases. The Bio Blast platforms are based on deep understanding of the disease-causing biological processes, and potentially offer solutions for several diseases that share the same biological pathology. For more information please visit the Company's website, www.bioblast-pharma.com, the content of which is not incorporated herein by reference.

Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients (HOPEMD)

This study is currently recruiting participants. (see Contacts and Locations)
Verified November 2014 by Bioblast Pharma Ltd.
Sponsor:
Information provided by (Responsible Party):
Bioblast Pharma Ltd.
ClinicalTrials.gov Identifier:
NCT02015481
First received: December 8, 2013
Last updated: November 16, 2014
Last verified: November 2014
  Purpose
The Purpose of this study is to assess the Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Oculopharyngeal Muscular Dystrophy (OPMD) Patients.

ConditionInterventionPhase
Oculopharyngeal Muscular DystrophyDrug: CabalettaPhase 2

Study Type:Interventional
Study Design:Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title:Multi-Center, Dose-Escalation Study, to Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta® in OPMD Patients

Resource links provided by NLM:

Sunday, November 16, 2014

Any physical activity benefits your brain

This study observes how any physical activity has positive benefits for adult brains. -JR

Everyone knows that exercise makes you feel more mentally alert at any age. But do you need to follow a specific training program to improve your cognitive function? Science has shown that the important thing is to just get moving. It's that simple. In fact, this was the finding of a study conducted at the Institut universitaire de gériatrie de Montréal (IUGM), an institution affiliated with Université de Montréal, by Dr. Nicolas Berryman, PhD, Exercise Physiologist, under the supervision of Dr. Louis Bherer, PhD, and Dr. Laurent Bosquet, PhD, that was published in the journal AGE (American Aging Association) in October.
The study compared the effects of different training methods on the cognitive functions of people aged 62 to 84 years. Two groups were assigned a high-intensity aerobic and strength-training program, whereas the third group performed tasks that targeted gross motor activities (coordination, balance, ball games, locomotive tasks, and flexibility). While the aerobics and strength-training were the only exercises that led to physical fitness improvements after 10 weeks (in terms of body composition, VO2 max, and maximum strength), all three groups showed equivalent improvement in cognitive performance.
The subjects in the third group performed activities that can easily be done at home, which is excellent news for sedentary people who can't see themselves suddenly going to a gym to work out. To improve your cognitive health, you can simply start by doing any activity you like.
"Our study targeted executive functions, or the functions that allow us to continue reacting effectively to a changing environment. We use these functions to plan, organize, develop strategies, pay attention to and remember details, and manage time and space," explained Dr. Louis Bherer, PhD.
"For a long time, it was believed that only aerobic exercise could improve executive functions. More recently, science has shown that strength-training also leads to positive results. Our new findings suggest that structured activities that aim to improve gross motor skills can also improve executive functions, which decline as we age. I would like seniors to remember that they have the power to improve their physical and cognitive health at any age and that they have many avenues to reach this goal," concluded Dr. Nicolas Berryman, PhD.
Read more here

Saturday, November 15, 2014

What is a good Diet for Ataxia? Ensure that there is enough vitamin E !

I was asked recently, what is a good ataxia diet? Ataxia is caused by many disorders so its very hard to make useful AND general recommendations.

Here is a link to the National Ataxia Foundation Site's recommendations

But ask your doctor and don't overdo vitamins like vitamin E.

Here is one recent article on the topic
.  - JR




 2014 Feb 28;260:120-9. doi: 10.1016/j.neuroscience.2013.12.001. Epub 2013 Dec 14.

Vitamin E is essential for Purkinje neuron integrity.

Abstract

α-Tocopherol (vitamin E) is an essential dietary antioxidant with important neuroprotective functions. α-Tocopherol deficiency manifests primarily in neurological pathologies, notably cerebellar dysfunctions such as spinocerebellar ataxia. To study the roles of α-tocopherol in the cerebellum, we used the α-tocopherol transfer protein for the murine version (Ttpa(-/)(-)) mice which lack the α-tocopherol transfer protein (TTP) and are a faithful model of vitamin E deficiency and oxidative stress. When fed vitamin E-deficient diet, Ttpa(-/)(-) mice had un-detectable levels of α-tocopherol in plasma and several brain regions. Dietary supplementation with α-tocopherol normalized plasma levels of the vitamin, but only modestly increased its levels in the cerebellum and prefrontal cortex, indicating a critical function of brain TTP. Vitamin E deficiency caused an increase in cerebellar oxidative stress evidenced by increased protein nitrosylation, which was prevented by dietary supplementation with the vitamin. Concomitantly, vitamin E deficiency precipitated cellular atrophy and diminished dendritic branching of Purkinje neurons, the predominant output regulator of the cerebellar cortex. The anatomic decline induced by vitamin E deficiency was paralleled by behavioral deficits in motor coordination and cognitive functions that were normalized upon vitamin E supplementation. These observations underscore the essential role of vitamin E and TTP in maintaining CNS function, and support the notion that α-tocopherol supplementation may comprise an effective intervention in oxidative stress-related neurological disorders.
Copyright © 2013 IBRO. Published by Elsevier Ltd. All rights reserved.

KEYWORDS:

3-NT; 3-nitrotyrosine; AD; ANOVA; AVED; Alzheimer’s disease; CS; NPC; Niemann Pick disease type C; Purkinje neuron; SCA; TBARS; TTP; TTPA; Ttpa; US; analysis of variance; ataxiaataxia with vitamin E deficiency; cerebellum; conditioned stimulus; oxidative stress; spinocerebellar ataxia; t-HODE; thiobarbituric acid reactive substances; tocopherol; total hydroxyoctadecadienoic acid; unconditioned stimulus; vitamin E; α-tocopherol transfer protein; α-tocopherol transfer protein gene for the human version; α-tocopherol transfer protein gene for the murine version



Follow recommendations for healthy nutrtion

Friday, November 14, 2014

Did you know that TEXAS-REGULATED Health Plans MUST offer autism treatment benefits?

Smart -  I met a family that is buying a separate policy for thier child from UHC or Humana since their  out-of-state plan will not cover autism treatment.

The cheaper plan may not be less expensive at the end of the year.

 - JR

Perry Expands Autism Insurance Benefits in Texas

AUSTIN (June 15, 2013) -- Gov. Rick Perry has signed a bill expanding autism insurance benefits in Texas by eliminating any age caps for state-regulated health plans......expanding autism insurance benefits in Texas by eliminating any age caps for state-regulated health plans. In 2007, Perry signed legislation that made Texas just the third state nationally to enact autism insurance reform, then in 2009 signed another bill that raised the age cap from 5 to 9.

The new law eliminates the age 9 cap, but limits annual ABA benefits to $36,000 a year for children aged 10 and above. Under current law, state-regulated health plans are required to cover the diagnosis and treatment of autism, including behavioral health treatment, such as Applied Behavior Analysis (ABA), as well as speech, occupational and physical therapy.
Perry signed the bill without comment at the conclusion of the state's regular legislative session.
Sponsored by Senators Kirk Watson of Austin, Wendy Davis of Forth Worth and Eddie Lucio, Jr. of Brownsville, SB.1484 will take effect in September. To qualify for the extended coverage, children must be diagnosed with autism by the age of 10 to gain the coverage.
The House champions for the bill included Rep. Larry Gonzales of Round Rock, Rep. Ron Simmons of Carrollton, and Rep. Senfronia Thompson of Houston.
Texas is one of four states with existing autism insurance reform laws that has considered bills to expand coverage this year.

Sunday, November 09, 2014

Migraines in children from toddlers to teens

This article discusses migraine headaches and how they present in children from toddlers to teenagers.

A toddler is having bouts of projectile vomiting, quite frequently but not every day. Fearing something is "terribly wrong" with her daughter's digestive system, her mother takes her to the doctor. Within 20 minutes, the pediatrician has traced the girl's symptoms to motion sickness – she gets sick every time she rides in the car, or shortly after. Although she's never had a headache, by age 3 the girl is diagnosed with the neurological disorder called migraine.
What Is Migraine?
A simple definition is a “recurrent, episodic headache, or head pain, that typically lasts between two and 72 hours untreated,” says Andrew Hershey, chair and a professor of neurology at Cincinnati Children's Hospital Medical Center​​​​. For some people,​ migraine comes with nausea or vomiting, while others experience light and sound sensitivity. Headaches are usually throbbing and partial​​ (involving one side of the head) and moderately or severely intense.​ Migraine has a strong genetic component.
Hershey, director of the CCHMC Headache Center, says while most migraine episodes come and go, they can evolve into chronic migraine: “About a third of our patients get to the point that the headache just never leaves them.”
At least 10 percent of kids have migraine, Hershey says.​ While the rate is about 4 percent in preschoolers, by late teens it affects up to 15 percent of boys and 20 to 25 percent of girls. The youngest patient Hershey ever treated started at 10 months old.
What Triggers Migraine​?
“Migraine is more than a headache. It’s a generalized disturbance of function that involves brain and body,” says Joel Saper,​ a neurologist and director of the Michigan Head Pain and Neurological Institute in Ann Arbor.
Although many people believe stress "causes" migraine, researchers are still working to determine the complex causes for the condition. What's known is that children have a variety of migraine triggers that can set off an episode, including food sensitivities, fatigue, bright lights, loud noises and sleep changes.​
In younger kids, non-headache signs or “migraine equivalents” can include stomach pain, episodes of dizziness, vertigo (spinning), visual disturbances and sudden mood changes, Saper says. Motion sickness is migraine equivalent, he notes, and it’s been recently established that colic in babies is a forerunner to migraine.
Before starting any kind of treatment, it’s crucial to pin down the diagnosis. Some 300 other medical conditions cause headache in kids, Saper says. Emotional causes also have to be ruled out – like school avoidance in a kid who’s anxious or being bullied.
For parents, Saper says it’s key to find a knowledgeable health care provider, whether it’s a specialist, family practice doctor, nurse practitioner or physician assistant.
Life With Migraine for Kids
Two decades ago, Cathy Glaser ​was concerned about her 3-year-old daughter Samantha, who was having cyclical bouts of vomiting and upset stomach. Through careful questioning, the pediatrician traced the episodes to motion sickness.
Glaser knows a lot about migraine. “We like to say my daughter was doomed since both parents have migraine. It runs through both of our families,” says Glaser, who eventually started the Migraine Research Foundation in New York.
Although Samantha was stoic, Glaser could always tell when she had a migraine coming on. “I could look into my daughter’s eyes and see she was getting a headache,” she says. “I could look at her face – she was ashen. All of a sudden those sparkly child’s eyes were absolutely dead. And her whole affect changed.”
Migraine invaded every aspect of Samantha’s childhood, and she visited the emergency room several times a year. It hijacked family trips. On their way to a family reunion at Hersheypark in Pennsylvania, they were forced to pull off the New Jersey Turnpike, because Samantha was green with motion sickness.
School presents many challenges for kids with migraine. Glaser says that as a parent, you need to be your child’s advocate. And you have to be proactive.
“I used to go to school every year at the beginning, or before, with a doctor’s note about migraine and my own daughter’s treatment explain when she raises her hand and says, ‘I need to take my medication now,’ they need to let her go,” she recalls.
Samantha “didn’t want to miss her life,” Glaser says. Even as an 8-year-old, she would say “Mom, I can be alone and feeling horrible, or I can be at school with my friends feeling horrible. I choose school.”
Every summer Samantha's parents sent her off to sleep-away camps, ​and every time they got calls in the middle of the night to come get her – the camp couldn’t handle it. “This is the life of a child who has regular headaches who insists on living the life of a kid,” Glaser says.
Sameness Helps
Boys and girls get headaches equally until age 12 or 13 – but when girls start their periods, the picture changes. Migraine is an “estrogen-vulnerable” disorder, Saper explains. Hormone cycling in girls and later women – from menstruation, oral contraceptives and menopause – can make migraines worse. Boys are more likely to grow out of migraines than girls.
As she entered her teens, Samantha developed menstrual migraine. (She also gets weather headaches, when there’s a change in barometric pressure or a storm’s coming on.)
With migraine, “Sameness helps,” Saper says. Sleep is a big issue, and regular bedtimes and waking times are important. If kids have trouble sleeping, he says melatonin “is a particularly good medicine” to help.
Some children with migraine are food sensitive. “What they eat may make a difference, whether it be the gluten, cheese or dairy products – there’s a long list,” Saper says. Kids shouldn’t miss meals or skip breakfast.
Dehydration can also trigger migraines, and healthy habits include drinking plenty of water or other non-caffeine drinks. Exercise also helps, and so do relaxation and stress-management techniques
Not Your Parents’ Headache
For migraine that goes beyond a headache or two a month, research suggests the best approach combines medication and cognitive behavioral therapy. The two main drug categories are medications for treatment and for prevention.
NSAIDs such as Aleve and Ibuprofen are used to break acute headaches in kids, Hersey says ­– aspirin should be avoided because of the risk of liver problems. And parents and patients should be alert to overuse. For prevention, the drugs amitriptyline and topiramate are most commonly prescribed.
At Cincinnati Children’s Headache Center, after kids undergo a neurological and headache exam, the team comes up with a multipronged treatment plan, including medication choices and learning healthy habits. Kids with chronic headaches return for cognitive behavioral therapy, which takes about six weeks to absorb.
Part of the goal is getting kids to accept that they have a chronic illness and teaching them to manage their own care, Hershey says: “We often say to the kids, ‘It’s your headache – it’s not your mom and dad’s.”
Scott Powers, a pediatric psychologist at the center, is working with his team to fine-tune a migraine app that kids can use to track their episodes and symptoms electronically. The app also allows researchers to better connect the dots between triggers and headache timing.
College and Migraines
With the importance of regular sleep, healthy eating habits and keeping stress on an even keel – what could possibly go wrong when kids with migraine leave for college?
But when the time came, Samantha insisted on going away to a big school, Glaser says. After much discussion they agreed that Samantha would go to a school within driving distance, near a hospital and with a well-staffed campus health center.
“Her view about college was ‘I want to stop being 'Migraine Girl,'" Glaser recalls her daughter saying. “’I want to stop being defined by my disease.’” 
Read more here

Insomnia raises risk of fatal injury

Having insomnia can raise a person's risk of death by motor vehicle and other fatal injuries.

New research suggests that insomnia is a major contributor to deaths caused by motor vehicle crashes and other unintentional fatal injuries. The results underscore the importance of the "Sleep Well, Be Well" campaign of the National Healthy Sleep Awareness Project.
Results show that the risk of unintentional fatal injury increased in a dose-dependent manner with the number of insomnia symptoms present. People with all three symptoms of insomnia were 2.8 times more likely to die from a fatal injury than those with no insomnia symptoms, even after adjusting for potential confounders such as alcohol consumption and daily use of sleep medication.
Among the three insomnia symptoms, difficulty falling asleep appeared to have the strongest and most robust association with fatal injuries. People who almost always had difficulty falling asleep were more than two times more likely to die from a motor vehicle injury (hazard ratio = 2.40) and more than 1.5 times more likely to die from any fatal injury (HR = 1.66) than people who never had trouble initiating sleep. Further analysis found that self-reported difficulty falling asleep contributed to 34 percent of motor vehicle deaths and eight percent of all unintentional fatal injuries, which could have been prevented in the absence of insomnia.
"Our results suggest that a large proportion of unintentional fatal injuries and fatal motor vehicle injuries could have been prevented in the absence of insomnia," said lead author Lars Laugsand, MD, PhD, postdoctoral fellow in the department of public health at the Norwegian University of Science in Technology in Trondheim, Norway. "Increasing public health awareness about insomnia and identifying and treating people with insomnia may be important in preventing unintentional fatal injuries."
The study results are published in the November issue of the journal Sleep.
"Healthy sleep is essential for physical health, mental well-being, and personal and public safety," said American Academy of Sleep Medicine President Dr. Timothy Morgenthaler, a national spokesperson for the Healthy Sleep Project. "Sleep is a necessity, not a luxury, and the promotion of healthy sleep should be a fundamental public health priority."
Earlier this year the Healthy Sleep Project launched the "Sleep Well, Be Well" campaign to increase awareness of the importance of sleep as one of the three pillars of a healthy lifestyle. More details are available at http://www.projecthealthysleep.org.
The study involved that analysis of population-based survey data from 54,399 men and women between 20 and 89 years of age. Cause of death was identified using a national registry. During the 13-year follow-up period there were 277 unintentional fatal injuries, including 169 deaths from falls and 57 deaths from motor vehicle crashes.
According to the Centers for Disease Control and Prevention, there are more than 126,000 unintentional injury deaths in the U.S. each year, making it the fifth leading cause of death. There are more than 33,000 motor vehicle traffic fatalities and more than 27,000 unintentional fall deaths annually, as well as 29.3 million emergency department visits related to unintentional injuries.
Read more here

Sleep issues in children with mental health diagnoses

Sleep issues are common among children with mental health diagnoses, and may go undiagnosed due to the co-existing conditions.

Sleep difficulties, particularly problems falling asleep, are common among toddlers and preschoolers with mental health issues, according to a new study.
"Sleep problems in young children frequently co-occur with other behavioral problems, with evidence that inadequate sleep is associated with daytime sleepiness, less optimal preschool adjustment, and problems of irritability, hyperactivity and attention," said the study's leader, John Boekamp, clinical director of the pediatric partial hospital program at Bradley Hospital in Providence, R.I.
However, he said, sleep disorders may be unrecognized and underdiagnosed in young children, particularly when behavioral or emotional problems are present.
The study, published online in Child Psychiatry & Human Development, involved 183 children aged 6 years or younger receiving outpatient treatment for psychiatric problems. The researchers examined the prevalence of sleep disorders among these children and the nature of the sleep problems.
"The most common sleep difficulties reported nationally for toddlers and preschoolers are problems of going to bed, falling asleep and frequent night awakenings. Collectively, these problems are referred to as behavioral insomnias of childhood," said Boekamp in a hospital news release.
Recognized sleep disorders, particularly sleep onset insomnia, were more common than expected, the researchers found. Overall, 41 percent of children in the study met the criteria to diagnose a sleep disorder.
Sleep problems were most common in kids with disruptive behavior, and attention, anxiety and mood problems, the researchers found.
Early sleep problems could not only be the result of behavioral and emotional problems, but could also contribute to them, the researchers noted.
"Essentially, these young children might be caught in a cycle, with sleep disruption affecting their psychiatric symptoms, and psychiatric symptoms affecting their sleep-wake organization," said Boekamp. "It is important for families to be aware of how important sleep is to the behavioral adjustment and well-being of young children."
Sleep problems can complicate treatment for challenging behaviors, such as aggression and attention and mood problems. Daytime sleepiness and fatigue can make these problems even worse, the study's authors noted.
"This study is a great reminder that it's critical for mental health providers working with young children and their families to ask about children's sleep," said Boekamp.
"Simple questions about children's sleep patterns, including how long it takes a child to fall asleep at night and how frequently a child awakens after falling asleep, may yield important information that is relevant to clinical care, even when sleep problems are not the primary focus of treatment," he explained.
Read more here