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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…
According to a story from ANCA Vasculitis News, a recent study has found that a low-dose regimen of glucocorticoids that explicitly exclude the drug methyprednisolone can be a useful therapy…
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…
Stem cell therapy via intravenous injection lessened symptoms and prolonged survival of Gaucher disease, reports a study using a mouse model. The study was published in Human Molecular Genetics, and reported…
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 sectorpublishingintelligence.co.uk, the drug company INVENTIVA's lead product candidate, known as lanifibranor, has earned Fast Track Designation from the US Food and Drug Administration (FDA) as…
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…
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Pamela Smith, age 67, had run out of options when her melanoma metastasized and became inoperable. According to a recent article in MedicalXpress, in January 2014, Pamela joined the…
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…
October is Gaucher Disease Awareness Month! The National Gaucher Foundation (NGF) has some wonderful resources that can come in handy for the Gaucher patient community. Let's take a look below!…
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…
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The Providence Journal recently published an article by Brittany Ricci, a student at Brown University’s Alpert Medical School, detailing her year-long struggle to receive the treatment her doctor had prescribed…
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…
A new treatment was recently tested for blastic plasmacytoid dendritic cell neoplasm (BPDCN). A recent article in MD Anderson News points out that in the U.S., five hundred to…
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…
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