FGFR3-related chondrodysplasia

This study tests a daily oral drug called TYRA-300 in 92 children aged 3–10 with achondroplasia (a common form of dwarfism). The goal is to see if it safely increases their growth rate. Researchers will try different doses to find the best one. It's a Phase 2 trial, so it's still…

This study tests an oral drug called infigratinib in infants and children under 3 years old with achondroplasia, the most common form of dwarfism. The goal is to see if it safely improves growth and body proportions. The trial has several phases to find the right dose and then co…

This study tests a new medicine called BMN 333 against an existing drug (vosoritide) to see if it can safely improve growth in children with achondroplasia, the most common form of dwarfism. About 160 children aged 2 to 17 will take part. The goal is to measure how much they grow…

This study tests a weekly injection called navepegritide in 24 teenagers (ages 12-18) with achondroplasia, the most common form of dwarfism. The goal is to see if it safely increases growth rate over one year compared to a placebo. The treatment aims to manage the condition by im…

This study tests a weekly injection called TransCon CNP in infants under 2 years old with achondroplasia, the most common form of dwarfism. The goal is to see if it safely improves growth over 52 weeks. About 72 babies will take part, with some receiving the drug and others a pla…

This study tests a daily injection called vosoritide in infants and toddlers (0 to 3 years old) with hypochondroplasia, a genetic condition that causes short stature. The goal is to see if the drug safely improves growth over 52 weeks. About 60 children will receive either vosori…

This study looks at whether doing breathing exercises before orthopedic surgery (like knee, hip, or fracture repair) can help your lungs work better after the operation. It involves 36 adults who have used tobacco and are at risk for breathing problems after surgery. Participants…

This study tests whether a program of exercise and ergonomics training can reduce muscle and joint pain in teachers. About 80 teachers who have had pain for at least three months will be randomly assigned to receive the training or not. Researchers will measure changes in pain, d…

This study tests a web-based app called IAMABLE that provides evidence-based rehabilitation strategies for people with chronic conditions like heart disease, arthritis, or neurological disorders. Fifty adults aged 45 to 75 will use the app for 4 months to set goals and learn abou…

This study tests whether extra support for case managers helps veterans leaving the military better manage pain and reduce risky substance use. About 1800 veterans will take part. The program focuses on non-drug pain treatments and counseling.

This study observes about 170 children and adults with achondroplasia in the United States over roughly 5 years. Researchers will collect health records and questionnaires to track growth, medical issues, surgeries, and quality of life. The goal is to better understand the condit…

This study aims to learn more about rare skeletal disorders by collecting medical records, blood samples, and genetic data from 100 participants. People with known or suspected bone conditions, as well as their healthy family members, can join either remotely or in person. The go…

This study translates and tests several health questionnaires—covering pain, arm and leg function, mood, and daily activities—for use with Albanian-speaking people. Researchers will check if the translated versions are reliable and accurate by giving them to about 300 participant…

This study is creating a collection of blood, urine, and leftover surgical tissue from 350 patients with bone, calcium, and growth plate disorders. The goal is to better understand how these diseases progress and to find markers that could predict outcomes. No new treatments are …

This study tests a new tool called the PRISM Dashboard, which gives physiotherapists feedback on how their decisions compare to their peers. The goal is to see if this feedback helps reduce differences in care for people with muscle and joint pain. About 60 physiotherapists and t…

This study is creating a database of health information from people with achondroplasia, a common form of dwarfism. Researchers will collect growth measurements and surgery records from up to 1,500 patients across several U.S. hospitals. The goal is to answer future questions abo…

This study follows 252 adults with muscle, bone, or joint problems (like arthritis, back pain, or after surgery) to see how well they do their prescribed home exercises. Researchers will track who sticks with the program and why, looking at factors like pain, mood, and support. T…

This study tests whether using a virtual reality (VR) headset during shoulder exercises helps patients feel more engaged and motivated. Fifty adults with shoulder problems will do rehab exercises in a VR environment and then answer questionnaires about how easy and immersive the …

This study is creating a large database of genetic and physical information from 3100 children with craniofacial abnormalities, such as craniosynostosis and Pierre Robin sequence. Researchers will analyze this data to understand why these conditions vary so much from person to pe…

This study is for people aged 3 and older with achondroplasia or hypochondroplasia (types of skeletal dysplasia). It uses a wearable device called Syde® to measure limb movements over a year. The goal is to find out which movement measurements are reliable and useful for future r…

This study aims to create a secure national database for muscle and joint (musculoskeletal) conditions by collecting routine data from community clinics and GP practices. About 22,000 adults will have their information anonymously gathered to help develop a dashboard that tracks …

This study watches 2000 people having hip or knee replacement surgery to see how long spinal anaesthesia lasts. The goal is to learn how the amount of anaesthetic affects timing, helping doctors decide if patients can safely go home the same day. No new treatments are tested—just…

This study looks at how changing the heel cushion on an ankle-foot brace (AFO) affects walking in people who use one daily. Forty adults with leg injuries or nerve problems will try four different heel wedges—tall or short, soft or firm—while walking at controlled speeds. Researc…

This study looks at how 3D computer simulation and printing can help surgeons better plan and perform bone correction surgeries. Researchers will compare the planned corrections to the actual results in 100 children and young adults with rare bone deformities. The goal is to make…

This study follows 260 children with achondroplasia (ages 2.5 to 11) for up to 2 years to collect data on growth, medical complications, and quality of life. No treatment is given—it is purely observational to better understand the condition's natural course.

This study follows 4,000 children who receive orthopedic implants (like rods or screws) during standard care for bone fractures, deformities, or hip problems. Researchers will track how long the implants last, any complications, and how well they work over time. The goal is to ga…

This study is building a registry of people with rare bone diseases (skeletal dysplasias) in Italy. Researchers will collect medical history, genetic data, and treatment details over time to better understand how these conditions progress. No experimental treatments are given; th…

This study follows 144 adults with muscle or bone problems who are already scheduled to receive treatment with Chattanooga Intelect devices (like TENS or ultrasound). Researchers will measure pain, muscle strength, and movement range to confirm the devices are safe and work as ex…

This study watches children aged 2.5 to 17 with hypochondroplasia, a condition causing short stature, to track their growth and health over time. No treatments or drugs are given. The goal is to learn more about how the condition affects growth, body proportions, thinking skills,…