European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session was part of the 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was to have occurred…
Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe
Last week was Global Tracheostomy Tube Awareness Week. The mother of a child with complex health challenges started it. Her goals were to educate people and normalize her daughter’s trach. While Trach Week focuses on the use of a trach as a long-term treatment, it got me thinking about my…
Ofev, an approved treatment of idiopathic pulmonary fibrosis (IPF), may be considered as a monotherapy for patients who also have non‐small cell lung cancer and are unable to tolerate cancer chemotherapy, a case study reported. …
I am taking a deep breath as I announce to my beloved online community the following: I have COVID-19. I also have idiopathic pulmonary fibrosis (IPF). Until now, I thought this combination would be fatal, but I am here to tell you that this isn’t necessarily true. I’m a fairly…
Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. The 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was set for May 14–15 in…
The U.S. Food and Drug Administration (FDA) has designated AMP945, Amplia Therapeutics’ investigational anti-fibrotic therapy for idiopathic pulmonary fibrosis (IPF), an orphan drug, supporting its development, the company announced. Its decision came about one month after AMP945 also received orphan drug status as a potential pancreatic cancer…
Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. The 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was set for May 14–15 in…
Repeat hospitalizations for respiratory problems in people with chronic idiopathic interstitial pneumonia (c-IIP) — a group of chronic lung diseases that also include idiopathic pulmonary fibrosis (IPF) — are associated with poor long-term survival, a study reported. The study, “Characteristics of patients with chronic idiopathic interstitial…
The French Medicines Agency has authorized Genkyotex to launch a Phase 1 trial to evaluate the safety and pharmacological properties of high-dose setanaxib, the company’s investigational oral treatment for idiopathic pulmonary fibrosis (IPF). Setanaxib (formerly known as GKT831) is an investigational anti-fibrotic therapy that is thought…
Dear Christie, It’s 10 p.m., Dec. 26th, 2018. You just got this crazy phone call from Mom. She told you that her disease — which you still don’t know the name of — has a “cure.” You knew she…
