Overview

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We’d like to express our appreciation for the patients who have previously participated in our clinical trials. Your contributions will help others as a result of the knowledge gained from your participation.

Idiopathic Pulmonary Fibrosis

Idiopathic Pulmonary Fibrosis (IPF) is a chronic, progressive, debilitating, and usually fatal interstitial lung disease that affects approximately 100,000 people in the U.S. This orphan disease results in fibrotic scarring of the lungs. Current treatment options are limited and poorly tolerated.

Idiopathic means “of unknown cause”, though there are certain risk factors that are associated with a higher incidence of IPF, including:

  • Age (> 50)
  • Male gender
  • Smoking
  • Acid reflux
  • Family history of IPF

While many patients do not have symptoms early in the course of the disease, as IPF progresses, symptoms can include:

  • Persistent dry cough
  • Shortness of breath, especially with exertion
  • Chest pain
  • Loss of appetite and non-intentional weight loss
  • Fatigue
  • Swelling in the legs

There are 2 FDA-approved drugs to treat IPF. While both drugs have been shown to decrease the rate of loss of lung function, neither has demonstrated an impact on improved survival and both are poorly tolerated by many patients.

Source: NIH

Nonalcoholic Steatohepatitis

Although NASH is one of the most common causes of chronic liver disease in in the U.S., it is a disease that is unfamiliar to many Americans.  Despite this, its prevalence is increasing and by some estimates 27 million Americans will be living with NASH by 2030.  

NASH is a progressive condition that starts with an excess buildup of fat in the liver, leading to inflammation and liver damage. While the cause of NASH is unknown, it is associated with a broader set of metabolic disorders and important risk factors include elevated triglyceride or cholesterol levels, type 2 diabetes, high blood pressure and obesity, particularly with body fat concentrated around the waist.  NASH is also more prevalent in certain ethnic groups including Asian and Hispanic populations.  While there are generally no symptoms until late in the disease process, the initial inflammation often leads to fibrosis (scarring) of the liver, which can progress to cirrhosis (advanced, late-stage scarring).  Patients who develop cirrhosis are at risk for complications including liver failure and liver cancer (hepatocellular carcinoma). There is currently no approved treatment.  

Source: Drew, L. Fighting the fatty liver. Nature 550, S102–S103 (2017)
https://doi.org/10.1038/550S102a

Obesity

Obesity impacts more than 40% of U.S. adults and is a major risk factor for a variety of other serious diseases, including heart disease, stroke, type 2 diabetes, NASH and certain types of cancer.

Acute Respiratory Distress Syndrome

Acute Respiratory Distress Syndrome (ARDS) occurs when fluid builds up in the tiny, elastic air sacs, or alveoli, of the lungs, resulting in poor blood oxygenation. ARDS is typically a complication of some other primary condition, such as pneumonia, sepsis, or trauma. Current treatment is primarily supportive, along with treatment of the underlying infection or trigger. Mortality rates are high and many patients that survive experience lasting lung damage.

These links are meant for informational purposes only and are not meant to replace medical advice.