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…
According to a story from healthbeat.spectrumhealth.org, Tasha Veinbergs was 13 years old when she was first diagnosed with Pompe disease. She was visiting the doctor to treat a small cyst…
When you have a rare disease, you know that the way it effects your life goes far beyond the symptoms listed on WebMd. It riddles through plans for weekend, your…
According to a story from American Nurse Today, the US Food and Drug Administration (FDA) approved a treatment on September 6th, 2019 for interstitial lung disease associated with scleroderma, a…
According to a story from Angioedema News, the UK's National Institute for Health and Clinical Excellence (NICE) has officially recommended the drug lanadelumab (marketed as Takhzyro) for coverage under the…
A study record from clinicaltrials.gov is investigating the appearance of lung problems in patients with disorders affecting connective tissue. While prior research has documented the appearance of interstitial lung disease…
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