Diseases
Today, Insmed’s primary area of focus is on patients with nontuberculous mycobacterial (NTM) lung disease, a rare and serious disorder that can significantly increase morbidity and mortality.
Mycobacterium avium complex (MAC) is the group of bacteria that most commonly causes NTM lung disease. Mycobacterium abscessus, the second most common cause of NTM lung disease, is a particularly difficult-to-treat bacteria.
Nontuberculous Mycobacterial (NTM) Lung Disease
Patients with NTM lung disease can experience a range of symptoms that often worsen over time, including chronic cough, dyspnea, fatigue, fever, weight loss, and chest pain. In some cases, NTM lung disease can cause severe, even permanent damage to the lungs, and can be fatal.1,2Non-Cystic Fibrosis Bronchiectasis (NCFBE)
Non-cystic fibrosis bronchiectasis (NCFBE) is a chronic pulmonary disorder in which the bronchi become permanently dilated due to a vicious cycle of inflammation and complications from prior infections.3,4 NCFBE is marked by frequent pulmonary exacerbations requiring antibiotic therapy and/or hospitalizations. Symptoms include chronic cough, intermittent hemoptysis, increased sputum production, impaired mucociliary clearance, and frequent bacterial infection.3,4,5 Patients who experience frequent exacerbations are at risk of significant morbidity and mortality.
Pulmonary Arterial Hypertension (PAH)
Pulmonary arterial hypertension (PAH) is a serious, progressive, rare disease involving high blood pressure in the pulmonary arteries, which carry blood from the heart to the lungs.6,7 Signs and symptoms include shortness of breath, especially during activity, chest pain, dizziness/fainting, fatigue, and weakness.7 In addition to a significant symptom burden, progression of the disease leads to progressive heart failure.6,8 Patients with PAH have a 15% one-year mortality rate.
References
- 1 Park HY, Jeong B-H, Chon HR, Jeon K, Daley CL, Koh W-J. Lung function decline according to clinical course in nontuberculous mycobacterial lung disease. Chest. 2016;150(6):1222-1232.
- 2 Young JD, Balagopal A, Reddy NS, Schlesinger LS. Differentiating colonization from infection can be difficult. Nontuberculous mycobacterial infections: Diagnosis and treatment. J Respir Dis. 2007;28(1):7-18.
- 3 Prevalence and incidence of noncystic fibrosis bronchiectasis among US adults in 2013 (Weycker)
https://www.ncbi.nlm.nih.gov/pubmed/28555504. Accessed February 2019. - 4 Advances in bronchiectasis: endotyping, genetics, microbiome, and disease heterogeneity (Flume).
https://www.ncbi.nlm.nih.gov/pubmed/30215383. Accessed February 2019. - 5 American Lung Association. Bronchiectasis Symptoms, Causes & Risk Factors.
https://www.lung.org/lung-health-and-diseases/lung-disease-lookup/bronchiectasis/symptomscauses-
risk-factors.html. Accessed February 2019. - 6 National Organization for Rare Disorders. Pulmonary Arterial Hypertension.
https://rarediseases.org/rare-diseases/pulmonary-arterial-hypertension/#a_ected-populations. Accessed February 2019. - 7 Rare Disease Report. Rare Disease Quick Facts – Pulmonary Arterial Hypertension (PAH). https://www.raredr.com/news/rare-disease-quick-facts-pah. Accessed February 2019.
- 8 WebMD. Pulmonary Arterial Hypertension. https://www.webmd.com/lung/pulmonary-arterial-hypertension#1. Accessed February 2019.
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