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Bioscience Companies in Ohio Take Aim at Rare Diseases
According to a story from BioPortfolio, Rare Disease Day, which took place on February 28th, 2019, serves as a time of reflection on the progress that has been made in…
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The Ability to Study Individual Cells is Leading to New Scientific Discoveries
According to a story from nbcnewyork.com, scientific research on cells was always a challenging task. Trying to study a certain trait used to mean looking at a tissue sample, which…
EU Establishes United Repository for Rare Patient Data and Standards for Collection
Each year, Rare Disease Day (February 28th) incites individuals and organizations to investigate and reform their rare disease efforts. This year, the EU Commission has made drastic changes which should…
New Hope for Rare Disease Patients
Rare disease isn't as rare as it sounds. Ten percent of Americans have a rare disease-- and there are almost 7,000 known rare diseases. A rare disease, also known as…
Tiffany Senter’s Cystic Fibrosis Story: Gaining Strength from Idol Marilyn Monroe
Cystic Fibrosis Cystic fibrosis (CF) is a rare condition that causes mucus to build up in the lungs and the digestive system. It results in chronic lung infections, lung inflammation, and…
Phase 3 Study in Europe Shows Continued Positive Results for Potential New Cystic Fibrosis Treatment
Cystic Fibrosis Cystic fibrosis (CF) is a rare disease caused by the lack (or malfunctioning) of the cystic fibrosis transmembrane conductance regulator (CTFR) protein. This occurs when children inherit a mutated…
NanoPODS: A New Way to Effectively Deliver Treatment in Cystic Fibrosis?
The Problem One of the greatest challenges in the investigation of nanoscale treatments for people with rare diseases is figuring out how to administer the therapy to the correct part…
Cystic Fibrosis Hearing Loss Drug Aims for Phase 2 this Summer
According to an article from Pharmaceutical Daily, the Seattle-based drug company Sound Pharmaceuticals is set to soon begin a phase 2 clinical study for a cystic fibrosis (CF) drug candidate…
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European Commission Approves Label Extension of Cystic Fibrosis Drug
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…
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What Can be Done to Resolve Shortages of Donated Organs for Transplant?
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…
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MassBio President Knows Difficulty of Living with Cystic Fibrosis
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…
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New Updates From Translate Bio in Drug Development for Cystic Fibrosis and OTC Deficiency
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…
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Cystic Fibrosis Patient and Advocate Celebrates Her First Year Following Lung Transplant
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…
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Patient Centricity Helps Facilitate The Introduction of New Therapies for Rare Diseases
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…
Phase 3 Clinical Trial has been Initiated for Dermatomyositis
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,…
4 Facts You Need to Know About Cystic Fibrosis
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)…
New Algorithm May Diagnose Cystic Fibrosis Faster than Physicians
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…
How Planning a Wedding Solved the Riddle of a Sister’s Mysterious Illness
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…
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Blood Test Could Help Bring Personalized Treatment for Cystic Fibrosis
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…
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Three Drug Combination for Cystic Fibrosis Performs Well in Clinical Trial
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…
This Just In: India Never Implemented their Rare Disease Policy as Promised
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…
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UK Cystic Fibrosis Patients are Protesting to get a Vital Drug Covered on the NHS
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…
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Is Phage Therapy the Best Hope for Cystic Fibrosis Patients That Can’t be Treated With Antibiotics?
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…
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Study Finds That Cystic Fibrosis Patients Aren’t Getting the Treatment They Need to Fight Infection
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…
<a href="https://pixabay.com/users/Pexels/">Pexels</a> / Pixabay
This Cystic Fibrosis Patient’s Story Highlights the Limitations of Clinical Trials
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…
