(BPT) - This article was sponsored and developed by Neurocrine Biosciences, Inc. Cerebral palsy (CP) is a neurodevelopmental disorder affecting movement and posture that begins in early childhood development. It is the most common cause of motor disability in children, affecting about one in 345 children in the United States. 1-2 It may happen as a result of lack of oxygen during or around birth, stroke, infection, a problem with metabolism or other problems that cause injury or affect the development of parts of the brain involved in movement control in the first few years of life. 3 CP is a permanent condition, affecting a person for their entire life. People living with CP typically have motor problems, which may include spasticity (abnormal muscle tightness), dyskinesia (uncontrolled movements) or ataxia (poor muscle control), and many people have a mix of these motor disorders. 4 Dyskinesia due to CP (DCP) is one of the most disabling forms of CP and impacts approximately 10% to 20% of people living with CP. 5-6 According to the Cerebral Palsy Foundation (CPF), the combination of irregular and unpredictable movements (chorea) and twisting and repeating movements (dystonia) often disrupt activities and cause significant functional impairment, including the ability to maintain balance, walk or fine motor control. 7 Heather Riordan, M.D., Neurologist and Movement Disorders Specialist, Director of the Phelps Center for Cerebral Palsy at the Kennedy Krieger Institute, discusses the symptoms of chorea and impact on patients in a video shared on the CPF website here . About 30% of people with DCP are affected mostly by these involuntary and irregular movements (chorea), while dystonia is the predominant motor finding in the remaining 70%. 8 However, it is common for both chorea and dystonia movements to be present together. 6 For people living with DCP, these movements can occur in any region of the body, including the arms and legs, torso and face and may vary in severity. They are often triggered or made worse by stimulation or stress. Because dyskinesia can occur at rest and/or when actively using the body, the problems with movement are very burdensome in day-to-day living, with discomfort and pain affecting the quality of life for people and their caregivers. 9 Jen Lyman, mom to a son with DCP, highlights how dyskinesia makes communicating more difficult. "The most difficult thing about dyskinesia is watching my son struggle to do things that he wants to do, but the extra movements get in the way...special things, such as using a touch screen to video chat with his grandmother, are nearly impossible despite his best efforts to use his hands and fingers. Something so simple, yet so special for him should be effortless." DCP has a wide-ranging impact on the individual, including lifelong challenges with movement, a higher risk of other medical issues, a higher rate of mental health challenges and difficulties with communication. 10-12 "Those of us who have the privilege to serve patients with dyskinetic, hyperkinetic or mixed cerebral palsy see the functional impact of this very challenging type of tone every day," said Susan Biffl, M.D., Rehabilitation Medicine Specialist at Rady Children's Hospital-San Diego and Assistant Professor at UC San Diego School of Medicine. "Patients face challenges with self-care, independence, mobility, communication, fine motor skills that affect occupational and recreational activities, swallowing, social interactions and even sleep. As this tone is variable, it is much more challenging to treat than more consistent tone issues, such as spasticity." Treatment Options There are currently no Food and Drug Administration-approved treatments for DCP. Dystonia and chorea are often managed separately as they respond differently to various treatments, which can make treatment challenging. 3 Most drugs being investigated for DCP have focused on dystonia, and more research is needed to determine potential treatments for chorea. New treatment options are needed to help manage these movement disorders and improve daily function and quality of life for those affected. "After 20 years of caring for individuals with cerebral palsy, I continue to find those with dyskinetic cerebral palsy among the most challenging to treat, largely due to the limited effectiveness of available pharmacologic options," said Joyce Oleszek, M.D., Professor, Department of Physical Medicine and Rehabilitation, University of Colorado School of Medicine. "There is an urgent need for more robust evidence to support pharmacologic treatments for this condition, given its profound impact on function, comfort and quality of life." Ongoing Research Clinical studies are important in the development of treatment options, allowing researchers to evaluate the safety and effectiveness of new medicines. The data from clinical studies are used to determine if an investigational treatment can be approved for use to treat certain disorders. There are ongoing clinical studies evaluating potential treatments for DCP, including one for chorea. Neurocrine Biosciences is conducting the KINECT ® -DCP clinical study, a Phase 3, randomized, double-blind placebo-controlled study investigating the study drug, valbenazine, for the treatment of DCP. The clinical study is currently enrolling children, adolescents and adults six to 70 years of age who have been diagnosed with DCP. "The Cerebral Palsy Foundation is excited to partner with Neurocrine Biosciences on this study," said Rachel Byrne, Executive Director of the Cerebral Palsy Foundation. "We are proud to support strong research that can potentially bring meaningful change to the lives of those living with cerebral palsy." If you or someone you know may be eligible for this research study, please visit the study website for additional information here: FindADCPStudy.com . REFERENCES © 2024 Neurocrine Biosciences, Inc. All Rights Reserved. CAP-NBI-US-0115 12/2024 NBI-98854-DCP3018_11DecMatRelease_v1.0_25November2024
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As snow blankets the Colorado mountains, outdoor enthusiasts have a unique opportunity: the winter hut trip. Whether you’re a skier or a snowshoer, a hut trip offers an excellent opportunity to connect with nature while exploring the rugged beauty of the state’s wilderness and enjoying the warmth and camaraderie of a cozy mountain hut. From the towering peaks of the San Juan Mountains to the snow-covered trails in Summit County, Colorado’s winter hut trips provide an unforgettable experience for adventurers of all levels. Huts fill up fast, so check each property’s for pricing and availability. Located between Telluride and Silverton near the top of Ophir Pass in the San Juan Mountains, the Opus Hut was built for backcountry skiers, mountaineers, hikers and mountain bikers. At 11,700 feet, the hut sits at treeline with low-angle glades below and open slopes above. While intermediate powder skiing is available out the back door of the hut, owner Travis Mohrman said the terrain is best suited for experienced backcountry skiers. Mohrman estimates that 15% to 20% of the groups visiting Opus Hut do so with guides. “They’re not personally comfortable with the terrain or they’re not from the area,” Mohrman said. “They guides are knowledgeable about local conditions — what the snow is, what’s safe and what’s not safe.” The cabin accommodates up to 20 people in five rooms. Some visitors book the whole hut and bring friends and family, while others reserve available beds in unbooked rooms. The hut features solar-powered lighting and 110-volt outlets for charging electronic devices. It also has filtered drinking water, hot and cold tap water, and indoor composting toilets. It provides full bedding and clean sleeping bag liners. During winter, the hut has four to six employees who sleep in a separate cabin. They prepare meals with natural, organic, and, when possible, locally grown products. The hut accommodates vegetarian, vegan, and gluten-free diets–just be sure to inform the staff beforehand. It also offers beer, wine, and a limited selection of spirits for purchase. “You can travel much lighter if you don’t have to bring in your food,” said Mohrman, who took over the hut three years ago. “You don’t have to focus on the upkeep of being in the backcountry.” Reservations for Opus Hut open Aug. 1. “The winter fills up quick,” Mohrman said. “Every winter weekend books in the first five minutes.” Nestled at 11,200 feet in the San Juan National Forest, Campfire Ranch Red Mountain Pass is the perfect base for exploring world-class skiing, split boarding, snowshoeing, and ice climbing. Located between Silverton and Ouray, it’s is accessible during the winter via a half-mile backcountry over-snow approach. Campfire Ranch is an ideal choice for novices. While other Colorado hut systems require you to carry your own food, bring sleeping bags, and live off-grid, this one provides food service, solar-powered electricity, Wi-Fi, and bedding. The dog-friendly cabin accommodates eight people. “We took a hospitality approach to remove barriers to entry for people who want to have the experience but don’t have the gear or the knowledge,” said Katrin Meiusi, director of marketing for the properties. Campfire Ranch first opened a campground on the Taylor River in Almont near Crested Butte. RVs are not permitted at the campground, which is open from May to October. Amenities include unlimited firewood, clean bathrooms, and drinkable well water. The 38 backcountry huts managed by the non-profit 10th Mountain Division Hut Association are connected by 350 miles of trails among some of the tallest peaks in the lower 48 states. All huts, some of which accommodate up to 17 people, have kitchens with propane burners for cooking — propane is provided. They provide pots, pans, potholders, dishware, cooking and eating utensils, a percolator or French press for coffee, salt and pepper, paper towels, dish soap, hand sanitizer, cleaning supplies and trash bags. Some huts have ovens and propane grills. All huts provide lighting from on-site solar power, propane or a generator. A few huts also have outlets for charging small devices such as phones. The huts have either an outhouse or an indoor bathroom with toilet paper supplied. All huts include mattresses and pillows, but you must bring your sleeping bag and pillowcase. Summit Hut Association operates five backcountry huts open for winter from November to May. Francie’s and Janet’s cabins are also open for summer use from July to September. All huts have solar-powered lights, fully stocked kitchens, and wood-burning stoves. Francie’s, Janet’s, and Sisters’ cabins have saunas and indoor toilets. The association hosts its annual Backcountry Ball fundraiser in October at The Maggie on Peak to kick off the season. The event includes dinner, drinks, a silent auction and entertainment. Proceeds help maintain the network of backcountry cabins.
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