While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene therapies to treat a variety of rare diseases. That’s the consensus of industry experts who spoke in a May 26 webinar…
COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say
Learning how to balance personal tasks with my health needs has been a difficult lesson since my diagnosis of idiopathic pulmonary fibrosis (IPF). It’s something I have not yet mastered. Previously, I sought recommendations from my healthcare team about how to do this better. They encouraged me to think of…
A real-world, multi-center study in Poland confirms the effectiveness and safety of two-year treatment with Esbriet (pirfenidone) for people with idiopathic pulmonary fibrosis (IPF). The study “A multicentre retrospective observational study on Polish experience of pirfenidone therapy in patients with idiopathic pulmonary fibrosis: the PolExPIR study” was published…
Ofev (nintedanib), an oral medication by Boehringer Ingelheim, is now approved to treat people with progressive fibrosing interstitial lung diseases (ILDs) in the U.S. and Canada. These approvals, by the U.S. Food and Drug Administration (FDA) and Health Canada, come on the heels of findings from the…
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…
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…
