United Therapeutics Sponsors PFF Registry


In a recent press release from mid-January 2020, the Pulmonary Fibrosis Foundation (PFF) announced that its registry, the PFF Registry, was being sponsored by biotechnology United Therapeutics. This sponsorship will allow researchers to understand the intersections between idiopathic pulmonary fibrosis (IPF) and pulmonary hypertension (PH), or high blood pressure in the lungs.

PFF Registry

According to the patient registry website, the PFF Registry:

is a valuable research tool available in the fight against PF. The registry contains information about how each [participant’s] diagnosis was made, symptoms from PF, medical test results, the use of medications used to treat PF, and even captures information about how people are doing years after enrollment.

Currently, enrollment for the PFF Registry is closed. However, the Registry contains data from 2,003 patients. 81.5% of these patients are participating in the biorepository. The Registry has followed patients for an average period of 26.2 months, or slightly over 2 years. Enrollment will re-open at some point in 2021, during which time caregivers, family members, and those who have received a lung transplant will also be able to contribute.

Goals of the PFF Registry include:

[collecting] medical information to support research that develops new ways to improve the quality of the lives of people with PF; [creating] a Registry of patients with PF for participation in additional research studies in the future; [and collecting] data on the patterns of clinical care delivered at PFF Care Centers, which can improve our understanding of the most effective ways to care for people with PF.

PFF and United Therapeutics

The PFF Registry is spearheaded by the Pulmonary Fibrosis Foundation. Founded in 2000 by Albert Rose and Michael Rosenzweig, the PFF is:

a 501(c)(3) nonprofit organization dedicated to identifying effective PF treatments and assisting those living with the disease. The brothers experienced firsthand the devastating effects of PF when their sister Claire passed away from the disease; [later] both brothers were also diagnosed with PF, and it was their vision and dedication that led to the creation of the Foundation.

Now, United Therapeutics is joining the PFF Registry as a sponsor. Additional sponsors include Genentech and Boehringer Ingelheim. United Therapeutics is working to address research and treatments for patients with rare pulmonary conditions. Currently, there are no FDA-approved treatments for patients with pulmonary fibrosis who also have pulmonary hypertension. Through this sponsorship, finding a treatment may be achieved.

Idiopathic Pulmonary Fibrosis (IPF)

The aptly named idiopathic pulmonary fibrosis (IPF) refers to the fibrosis, or hardening, of scar tissue in the lungs resulting from an unknown cause. Some doctors believe that IPF has an underlying genetic cause, while others believe the condition is a result from environmental triggers such as cigarettes, metal and wood dust, or viral infections. As lung tissue becomes fibrotic, oxygen is unable to enter the bloodstream, causing a host of health effects. An estimated 13-20 out of every 100,000 people globally have IPF. Following the diagnosis, the typical survival rate is around 3-5 years. Symptoms include:

  • Shortness of breath and/or difficulty breathing
  • Unintended weight loss
  • A dry, hacking cough
  • Pulmonary blood clots
  • Clubbed toes and fingers
  • Appetite loss
  • Gastroesophageal reflux (GERD)
  • Pneumonia
  • Lung cancer
  • Fatigue
  • Muscle and joint pain
Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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