According to a story from BioPortfolio, the biotechnology company Vertex Pharmaceuticals recently announced that the European Commission has approved a label extension for the company's cystic fibrosis drug Orkambi. Under…
According to a story from The Chicago Tribune, insufficient supplies of donated organs that could be used for transplant surgeries result in 33 deaths per day in the US. At…
According to the Boston Globe, the president of the Massachusetts Biotechnology Council has a personal stake in his continued advocacy for pharmaceutical development in his home state. Robert Coughlin has…
According to a story from globenewswire.com, the messenger RNA therapeutics company Translate Bio recently released the latest updates in regards to two of its leading product candidates for the treatment…
According to a story from the Irish Examiner, 31 year old Orla Tinsley is celebrating her first year of life following her life saving double lung transplant operation. This lung…
According to a story from Pharmaphorum, there are a number of new treatments that have been introduced or have seen major strides in development over the last year. More patient…
Dermatomyositis (DM) is a disease which causes muscle weakness and rashes on the skin. Immunosuppressive drugs are a common treatment prescribed for the condition. Patients may also be given methotrexate, rituximab,…
When my family friend's new girlfriend came to meet us, she made the best impression with her positive attitude and general ease in interacting with a large (and probably overwhelming)…
A diagnosis means everything for a rare disease patient. However, to process 200 patient records manually, it would take the most dedicated physician approximately 40 hours. That's one full work…
The Heritage Florida Jewish News reported a story about one sister's journey to her Gaucher Disease diagnosis, thanks to her older sister's wedding prep. What is Gaucher Disease? Gaucher disease…
According to a story from EurekAlert!, a team of researchers used a combination of microarray technology and blood tests in order to distinguish distinctive molecular signatures in a group of…
According to a story from Xconomy, the drug developer Vertex Pharmaceuticals recently announced that a three drug combination treatment for the rare lung disorder cystic fibrosis has performed well in…
We all got really excited last year when India implemented a new plan to benefit rare disease patients. It was called the National Rare Disease Policy. Basically, it put 12.86…
Cystic fibrosis patients in the UK are planning a protest on November 19th, 2018 with the goal of getting Orkambi, an important cystic fibrosis drug, covered on the NHS. This…
According to a story from Buzzfeed News, Paige Rogers was 21 years old when she was admitted to the hospital. She was having trouble breathing because of cystic fibrosis. Over…
According to a story from EurekAlert!, a recent study from the Children's National Health System has found that some cystic fibrosis patients are not getting sufficient doses of antibiotics in…
According to a story from statnews.com, Ella Balasa, who has cystic fibrosis, has been seeking to join a clinical trial in hopes of finding a new treatment that can help…
Growing up without a diagnosis is hard. In a world which frequents new scientific discoveries, you're still living as a medical mystery. When no one knows the answer, patients often…
A new study into a potential drug for cystic fibrosis has begun. The Phase Ib/IIa trial will investigate the drug POL6014 in up to forty patients who have cystic…
According to a story from pm360online.com, the specialty pharmaceutical company Santhera Pharmaceuticals has recently announced of its Phase Ib/IIa clinical trial that will test its experimental product POL6014. This therapy…
According to a story from the Toronto Sun, the Canadian Agency for Drug and Technologies in Health (CADTH) recently announced that it would not be recommending the funding of the…
In order to improve the lives of patients for any illness, we all know research is an essential step. We also know that the most accurate research is derived from…
According to a story from Mirror, a group of seven women who met each other through online support groups recently met in person for the first time. The women in…
With technologies like CRISPR, it is possible to imagine a world where genetic diseases fade into obscurity. Gene editing technologies may not have reached their potential yet, but many people…
According to a story from the Irish Times, Orla Tinsley was on the precipice of death before she received her lung transplant to treat her cystic fibrosis. She had to…
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