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…
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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. …
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…
Lung evaluations via a CT scan might help predict the mortality risk in idiopathic pulmonary fibrosis (IPF) patients more accurately than the decline in forced vital capacity, a study shows. The study, “Serial CT analysis in idiopathic pulmonary fibrosis: comparison of visual features that determine…
A significant proportion of people with idiopathic pulmonary fibrosis (IPF) were found to have the sleep disorder restless legs syndrome (RLS), compared to a control group, according to new research. The data also showed that having RLS lowered sleep quality in IPF patients. The study, “Restless…
The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people with rare diseases, their families, and caregivers. Survey questions cover a patient’s physical and mental health, supply of treatments, and access to healthcare, among other…
