News

‘Rare’ Documentary in Kickstarter Campaign to Raise $45K by Oct. 28

A crowdfunding campaign aims to raise $45,000 to support “Rare,” a documentary film featuring the struggles and achievements of people living with rare diseases and their families. Sweis Entertainment and Digital Cave Media launched the campaign — allowing filmmakers to finish producing and to release the documentary — on Kickstarter.

New Data-sharing Program Aims to Speed Innovation in Rare Diseases

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…

Study Findings Support Furtherance of GED-0507 for IPF

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…

Tony Award Recognizes Actress for Work as PF Advocate

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…

Syndax, Incyte Join Forces to Develop Axatilimab for IPF

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…