A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and promoting the standardization of new data collection. Launched during a virtual workshop in September, the U.S. Food and Drug…
New Data-sharing Program Aims to Speed Innovation in Rare Diseases
GED-0507, Nogra Pharma’s experimental therapy, significantly reduced the levels of pro-inflammatory and pro-scarring molecules, while suppressing the maturation of myofibroblasts — the main drivers of lung scarring (fibrosis) — to a similar or greater extent as two approved anti-fibrotic therapies. These findings, based on data from mice with induced…
Photo courtesy of Aishia Burnett Day 30 of 30 This is Aishia Burnett’s story: January 2015: Picture it: I was working full time, going to school part time, a mom to two high schoolers, happily married to my best friend. I got pneumonia and my doctor sent me for…
Friendships can change when chronic illnesses like idiopathic pulmonary fibrosis (IPF) enter the picture. Some might struggle to understand a friend’s illness, needs, and limitations. And the friend with the illness might change too, depending on how they cope. However, I believe true friendships can withstand these challenges, and even…
Photo courtesy of Emma Schmitz Day 29 of 30 This is Emma Schmitz’s story: I hadn’t planned on being a caregiver for my mom in my 20s, but it felt like the least I could do after the support she and my dad have given me throughout my life.
You may have noticed that September is Pulmonary Fibrosis Awareness Month. Here at Pulmonary Fibrosis News, we’ve been running a social media campaign that showcases patients and their loved ones in our community. Additionally, the Pulmonary Fibrosis Foundation runs a yearly campaign encouraging advocates to “let the world know”…
Julie Halston, a Broadway theater veteran and comedian, has received the 2020 Isabelle Stevenson Tony Award for her longtime volunteer efforts for the Pulmonary Fibrosis Foundation (PFF). Halston, who is part of the PFF board, has raised more than $2 million for the organization. She also helped to bring…
Photo courtesy of Kevin Schaefer Day 28 of 30 This is Kevin Schaefer’s story: Four years ago, I accepted a position at Bionews Services, the parent company of Pulmonary Fibrosis News. Having lived with a neuromuscular condition called spinal muscular atrophy my entire life, I already had a…
The dynamic between an ill person and the loved ones who care for them should extend beyond the caregiver providing and the sick person receiving. The relationship should go two ways — thus, a care partnership. I define a care partner based on a presentation given at the Pulmonary…
Syndax Pharmaceuticals and Incyte are forming a global partnership to develop and commercialize axatilimab, an investigational therapy for idiopathic pulmonary fibrosis (IPF) and other diseases characterized by excessive scarring. The companies are planning to launch a proof-of-concept Phase 2 clinical trial to test axatilimab in IPF patients…
