With advocacy organizations gaining traction, sometimes patients with rare diseases may have a handful of options to choose from. Some patients may be quick to join patient advocacy groups…
According to a story from The Guardian, Jeremy Corbyn, the leader of the UK Labour Party, announced a remarkable plan at the most recent Labour party conference that has the…
Innovation and Collaboration Macular telangiectasia type 2 (MacTel) is a rare eye disease that causes progressive vision loss as patients age. Paul Bernstein is a physician at the John A.…
According to a story from Financial Buzz, the biotherapeutics company Bellerophon Therapeutics Inc. has recently announced that it has earned Orphan Drug designation from the US Food and Drug Administration…
Consent is a concept back under discussion in modern conversations as we readdress what it means to cross the line. Consent is no longer seen in the black and white…
According to a story from Spectrum News a recent study revealed two potential therapies that could treat symptoms associated with Rett syndrome, a rare disorder. The study involved the creation…
Cystinosis Cystinosis is a rare disease that causes cystine, an amino acid, to build up in the body's organs. However, there is treatment that can prevent this accumulation. Normally, patients…
Ischemic cardiomyopathy is a type of dilated cardiomyopathy, in which the capability of heart to pump blood is reduced. This causes lack of blood supply to heart muscles which result…
Happy Thursday everyone! This week, we're sharing a video that will warm your heart, as well as an article about how to improve care in older Multiple Myeloma patients. We…
According to a story from medicalxpress.com, Dr. Nathan Hoot is an emergency medical physician, but his role as a doctor doesn't entirely end when he leaves the hospital because his…
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FierceBiotech recently reported that Helixmith Co., based in Korea and previously known as ViroMed, coordinated a Phase 3 clinical trial involving five hundred diabetic peripheral neuropathy (DPN) patients. The…
On May 29th, 2019, the International Fibrodysplasia Ossificans Progressiva Association (IFOPA) alongside other members of the fibrodysplasia ossificans progressiva community, sat down for an hour long 'listening session' with representatives…
According to a story from Scleroderma News, the results of a recent mouse study could have significant implications for the future of scleroderma treatment. In the study, scientists used a…
According to a recent press release from the Pennsylvania-based Marinus Pharmaceuticals, the Company is reporting positive results of its phase 2 study of "ganaxolone," an investigational GABAA modulator for patients…
A recent publication from Healio detailed some of the challenges immunocompromised individuals might face during flu season. Influenza is a common and sometimes serious public health concern around the world, and…
Most young people are familiar with vaping: nicotine or THC repackaged in a sleek and less stigmatized device. It is less obviously recognizable as destructive as cigarettes in part because…
According to a story from outsourcing-pharma.com, the company Firma Clinical Research, which provides health care services to patients in clinical trials, has been getting a lot of business lately. This…
According to a story from Healio, a recent study has determined that older patients with multiple myeloma, a rare cancer that affects plasma cells, routinely under-utilize supportive care resources as…
According to a story from newton.wickedlocal.com, the first time that mother Melissa Chaikof realized that something was wrong with her oldest doctor was many years ago. When a nearby door…
According to a story from news.yale.edu, Yale alum Jordan Plotner began experiencing his first symptoms of Ehlers-Danlos syndrome when he was a sophmore. He experienced episodes of brain fog and…
According to a story from finanzen.ch, the US Food and Drug Administration (FDA) recently approved the drug rituximab (marketed as Rituxan) as a treatment in child patients aged at least…
According to a publication from EurekAlert, a study recently published in the medical journal Nature Genetics suggests how certain immune cells express genetic activity similar to that of variations of the…
The National Organization for Rare Disorders (NORD) is hosting its 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit on October 21-22 in Washington, D.C. NORD is a great organization and…
Talk about a bittersweet symphony! Published on Yale University's online paper, recent grad Jordan Plotner -- who is living with Ehlers-Danlos syndrome (EDS) -- organized a virtual musical ensemble with other…
The FDA announced last week that it has approved Rituxan (rituximab)to treat granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA) in children 2 years of age and older in combination…
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