The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases. “The RareLaunch program is…
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A photo of a bespectacled young boy, his red baseball cap slightly askew as he enjoys time outside, will be featured on the front cover of an upcoming calendar in the “Same But Different” contest to raise awareness about rare disorders. “A Lovely Day Out in Kew Gardens,”…
The combination of CB5138-2 — an experimental peptide, or small protein — and Ofev (nintedanib) reduced symptoms of idiopathic pulmonary fibrosis (IPF) better than Ofev alone in a preclinical study. Together, CB5138-2 (developed by CohBar) and Ofev (an approved IPF therapy marketed by Boehringer Ingelheim)…
ARDS-003, Tetra Bio-Pharma’s investigational medication for the treatment of hyperinflammatory lung injury, showed an anti-inflammatory effect in pre-clinical studies using a mouse model of pulmonary fibrosis (PF) and could be considered for the treatment of COVID-19, the company announced. Apart from reducing inflammation, the therapy also lessened…
A decrease in health-related quality of life (HRQoL) in people with idiopathic pulmonary fibrosis (IPF) is mainly associated with respiratory and emotional symptoms, a study shows. Routine assessment of these symptoms is important, as they may be related to other ones affecting HRQoL, researchers say. The study, “…
A Phase 2 study of Haduvio (nalbuphine extended release tablets) to treat chronic cough in people with idiopathic pulmonary fibrosis (IPF) is again enrolling eligible patients, Trevi Therapeutics, the therapy’s developer, announced. The U.K. trial was placed on hold due to the COVID-19 pandemic, as IPF patients…
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…
A new way of assessing ultrasound, a non-invasive technique using sound waves to produce pictures inside of the body, may be effective in diagnosing idiopathic pulmonary fibrosis (IPF), a study reported. This approach was able to quantify lung scarring, a hallmark of IPF, in a study in rats, as well…
All patients in a Phase 1/2 trial examining two doses of AP01, an inhaled version of pirfenidone for the treatment of idiopathic pulmonary fibrosis (IPF), are switching to the higher dose based on the recommendation of a data safety monitoring board (DSMB). Avalyn Pharma, AP01’s developer, reported that…
A Phase 2 study of NP-120 (ifenprodil) for chronic cough associated with idiopathic pulmonary fibrosis (IPF) has reached 25% of its enrollment target, Algernon Pharmaceuticals, the therapy’s developer, has announced. “I am very pleased to report that we have reached 25% enrollment in the study,” Christopher J. Moreau, CEO…
