Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say

A majority of rare disease patients using telehealth during the COVID-19 pandemicĀ thought the experience positive, and many would like the option of continuing its use in future appointments, a series ofĀ surveys found. The surveys were conducted by theĀ National Organization for Rare DisordersĀ (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 diagnosingĀ 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 patientsĀ in a Phase 1/2 trial examining two doses of AP01, an inhaled version of pirfenidoneĀ 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 coughĀ associated with idiopathic pulmonary fibrosisĀ (IPF) has reached 25% of its enrollment target, Algernon Pharmaceuticals, the therapy’s developer, has announced. ā€œI am very pleased to report that we have reached 25% enrollment in the study,ā€ Christopher J. Moreau, CEO…

Oct. 10 was World Mental Health Day. I am a bit late to the party, but with a twice-monthly publishing schedule, this is my first opportunity to share a column inspired by Mental Health Day. In previous columns, I have discussed my mental health struggles and how I use…

A protein-coding gene called Spred2 may contribute to pulmonary fibrosis (PF) development, potentially representing a new therapeutic target, according to the results of a new study. Titled “Spred2-deficiency enhances the proliferation of lung epithelial cells and alleviates pulmonary fibrosis induced by bleomycin,” the study was…

I began hearing the term “toxic positivity” this year, and the pandemic has illuminated the term’s meaning for me. According to The Psychology Group in Fort Lauderdale, Florida, toxic positivity refers to ā€œthe excessive and ineffective overgeneralization of a happy, optimistic state across all situations.ā€ Since the beginning of…

Treatment with inhalable bone morphogenic protein 4 (BMP4) — a soluble signaling molecule involved in the regulation of cellular identity — restored lung regeneration and improved lung function in a mouse model of idiopathic pulmonary fibrosisĀ (IPF). The data showed that scar-forming cells in IPFĀ lungs suppress BMP signaling…

Esbriet (pirfenidone),Ā an approved treatment forĀ idiopathic pulmonary fibrosis (IPF), seems to be safe in IPF patients who also have non‐small cell lung cancer (NSCLC) when combined with cancer therapy. The finding is detailed in the study, ā€œSafety and effectiveness of pirfenidone combined with carboplatin‐based chemotherapy in patients…