Alnylam Pharmaceuticals has been developing lumasiran as a potential treatment for primary hyperoxaluria type 1 (PH1). This investigation began with ILLUMINATE-A, a clinical trial whose results should be announced by…
According to a story from EurekAlert!, a new mathematical approach pioneered by a team of scientists from New York University and Cincinnati Children's could greatly improve the analysis of scientific…
Personalized medicine is the latest craze in patient care. In essence, it refers to the development of treatment options which take into account the patient as an individual. It reduces…
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Familial Hypercholesterolemia (FH) is often misdiagnosed as plain old high cholesterol because of overlapping symptoms such as elevated lipid levels. According to a recent article in MedicalView, FH is three…
According to a story from Pulmonary Hypertension News, the drug developer United Therapeutics recently issued a statement in which the company announced that it was ceasing the development of esuberaprost…
Happy Thursday! We hope everyone's month is going well! This week, we're highlighting two stories about drug pricing and reimbursement. Next, we have an article on mastocytosis diagnoses and (yet…
Upcoming Conference The International Society for Mannosidosis & Related Diseases (ISMRD) serves as an international patient advocate for those living with glycoprotein storage diseases. These include alpha-mannosidosis, fucosidosis, mucolipidosis type…
According to a story from AJC, three year old Reid Peteet is one of over 550 kids that have been diagnosed with acute flaccid myelitis since 2014. 2018 saw a…
For years FibroGen has been investigating a drug called pamrevlumab. They believe it could be an effective treatment for Duchenne muscular dystrophy (DMD), pancreatic cancer, and idiopathic pulmonary fibrosis (IPF).…
It's one thing to cope with a new rare disease diagnosis. It's another entirely to cope with that diagnosis and then four years later learn that diagnosis is incorrect and…
Biophosphonates (BPs) are common treatments for osteoporosis and metastatic bone cancer. They help to prevent bone loss. Unfortunately, these drugs can have serious side effects. One of the most severe…
Danon Disease Danon disease (DD) is an extremely rare condition that is not very well understood in the medical community. It was first described in 1981, and it is named…
According to a story from CRWE World, the genetic medicines company Audentes Therapeutics has recently announced its intent to develop new therapies for myotonic dystrophy and Duchenne muscular dystrophy. These…
People living with rare diseases such as amyotrophic lateral sclerosis (ALS) or muscular dystrophy as well as those who have suffered from a stroke or spinal cord injury often experience…
According to a story from Xconomy, a recent report from the Institute for Clinical and Economic Review (ICER), based on Boston, is calling for reductions to the price tags of…
According to a recent article in Check Orphan, genetic and biochemical testing are combined in a new method of metabolic testing called the CentoMetabolic panel. The announcement comes from…
Mastocytosis Mastocytosis is a rare disease caused by mutations in the KIT gene. The condition causes mast cells to accumulate in the body. Depending on the location of these immune…
According to a press release from American biotechnology company Soligenix, the European Medicines Agency's (EMA) Pediatric Committee recently agreed to the Company’s Pediatric Investigation Plan (PIP) for experimental oral mucositis…
According to a press release from the Muscular Dystrophy Association published at Biospace, the group has resolved to award eight new research grants for the study of amyotrophic lateral sclerosis…
According to an article from Healio, a recent study found that there has been a significant increase in the rate of Parkinson's occurrence around the globe over the last 25…
Happy Spring! Anyone else got that spring fever? Unless you're getting tortured by allergies, it is a great time to be outdoors. The bees are buzzing, the flowers are blooming,…
Tracheostomies in ALS A tracheostomy is essentially an invasive procedure which helps patients breathe. It also removes secretions from their lungs. Doctors cut an opening into the patient's neck and…
Glioblastoma Glioblastoma is a rare cancer which begins in the brain. Approximately 20% of all tumors that occur in the brain are glioblastomas. These tumors are highly malignant because they…
RADOIR Rare Diseases Foundation of Iran (RADOIR) is an organization dedicated to supporting those living with rare diseases in Iran. It is a nonprofit which was founded in 2008 with…
A recent article in globenewswire.com news reported that the FDA has approved Fast Track Designation for AMT 130, an experimental gene therapy for the treatment of Huntington’s disease. The…
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