Pulmonary Arterial Hypertension (PAH)
Study to Evaluate the Efficacy and Safety of Once Daily Treprostinil Palmitil Inhalation Powder in Participants with Pulmonary Arterial Hypertension
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New frontiers in medical research
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Your next step helps guide the way forward
Enrollment in a clinical trial may give your journey more meaning. And while the path is personal, we know the process of choosing to participate in a clinical trial may be challenging. Learn more about trial opportunities and information to discuss with your care team – so you can explore what may be possible in clinical trials, together.
CURRENT AREAS OF RESEARCH
Insmed is committed to helping people with serious diseases. We’re currently conducting research in the following areas:
AT is a rare genetic disorder that affects the nervous system (helps all parts of the body to communicate with each other), immune system (helps the body to fight against infections and other diseases), and other body systems. It is caused by a change (mutation) in the gene that controls the speed at which cells grow and divide. Symptoms of the disease get worse over time and during the lifetime of the AT patient and may include lack of body balance, lack of coordination of body movements, difficulty speaking (slurred speech), difficulty swallowing, unintentional movement of body parts, difficulty coordinating eye movement, and a weak immune system.
ALS, also known as Lou Gehrig's disease, is a neurodegenerative disorder that affects motor neurons. Motor neurons are the nerve cells in the brain and spinal cord that control muscle movement. Early symptoms of ALS include muscle cramping and weakness, slurred speech, and difficulty chewing or swallowing. As a progressive condition, these symptoms tend to worsen over time.
DMD is a genetic condition that causes progressive muscle weakness. It primarily affects young males and is caused by a mutation in the gene responsible for producing dystrophin, a protein that helps keep muscles strong.
PH is a rare, progressive disease in which the arteries of the lungs become narrowed and stiff. This causes high blood pressure (hypertension) in these arteries.
For information on our research, visit our Pipeline
Latest Clinical Trials
Learn more about our current clinical trials. Be sure to talk to your doctor and care team to see if one is right for you.
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Ataxia Telangiectasia (AT)
Longitudinal Study Comparing the Natural History of Ataxia Telangiectasia in Children to Healthy Comparators
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Amyotrophic Lateral Sclerosis (ALS)
A Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal (IT) Injection of INS1202 in Participants With Amyotrophic Lateral Sclerosis (ALS)
View trial informationDuchenne Muscular Dystrophy (DMD)
A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)
View trial informationPulmonary Hypertension Associated with Interstitial Lung Disease (PH-ILD)
Study to Evaluate the Efficacy and Safety of Treprostinil Palmitil Inhalation Powder in Participants with Pulmonary Hypertension Associated with Interstitial Lung Disease
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For information on our trials, visit our Trials in Progress
As research opens new doors to finding ways to diagnose, prevent, treat, or cure disease and disability, clinical trial participation is essential to help us find the answers.
What’s in it for you?
Getting involved in clinical trials helps to advance our understanding of science and diseases. In addition, some patients who join may gain access to:
- Better condition understanding
Access to diagnostic tests and education from research team - Enhanced care and monitoring
Frequent and comprehensive checkups with detailed assessments - Thorough screening process
In-depth evaluations that may affect your condition management - Psychological or emotional value
A proactive approach to address overall health - Personalized support
Gained resources including clinicians and care team - Reimbursement for costs
Participation often includes covering study-related expenses
All clinical trials are different and experiences may vary.
It’s also important to keep these factors in mind when deciding whether to apply to a clinical trial:
- Convenience of the trial location
Participation could require significant travel to the nearest trial location - Medication may come with side effects
Early testing results may also uncover potential safety concerns - Effectiveness of the drug
Positive treatment results are not guaranteed for trial participants - Experimental treatment may not be given to participants
Those in the “control” group may be given a placebo instead of investigational drug - Significant time commitment
For trial participants as well as caregivers and loved ones that are involved - Impact on daily life
Participation may disrupt daily routines and activities
Ask your healthcare provider about clinical trials and learn more about patient advocacy groups that may be suited for you.
Testimonial from a clinical trial participant
“I was nervous when I decided to check out clinical trials for my condition, but I knew I needed something different.”
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