According to a story from BioSpace, the biopharmaceutical company Sobi and the biotechnology company NovImmune SA have recently announced that the US Food and Drug Administration (FDA) has approved Gamifant,…
According to a story from the biotechnology company Amgen, a review from the European Medicines Agency (EMA) has recommended that the indication for blinatumomab, marketed as BLINCYTO®, be expanded. The…
According to the US Centers for Disease Control and Prevention, there have been 10 more confirmed cases of acute flaccid myelitis (AFM), a polio-like illness that mostly affects children. This now…
In 2001, I developed severe chronic pain from a neurological movement disorder called dystonia. My life before and after dystonia are as different as night and day. I went from a very active lifestyle to…
The Newborn Screening Saves Lives Act was originally introduced to facilitate the spreading of awareness and education related to newborn disease screenings. The screening of newborns has had a major…
According to a story from Scimex, the Children's Medical Research Institute in Sydney, Australia, has recently agreed to a new partnership with LogicBio Therapeutics, a biotechnology company from Boston, MA.…
According to a story from rdmag.com, the biopharmaceutical company IntraBio Inc., recently announced that its IB1000 series of investigational therapeutic compounds have been granted Orphan Medicinal Drug Designation as a…
Eltrombopag (Promacta) has now been approved for all individuals in the US with severe aplastic anemia. The drug will be accessible to both pediatric and adult patients. This is huge news.…
According to a story from CNBC, 53-year-old James Addie has been living with hemophilia since the day he was born. Because hemophilia is a genetic disease, the blood disorder is…
We are entering the Thanksgiving season. I’ve been taught all my life that it shouldn’t be only a season, but an attitude year around. But how? By telling Mom thank…
According to a story from CheckOrphan, a wide array of experts are raising concerns about India's rare disease policy, which was first unveiled in May of 2017. Despite being hailed…
Glioma is a type of cancerous tumor that originates in the brain. It has various types and the tumors range in aggressiveness. There are around 11,000 people just in the…
The Jaiswal family of Lucknow, the capital of Uttar Pradesh state in northern India, knows firsthand that rare diseases are not always that. CheckOrphan has reported that three of his…
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…
According to a story from The Oncology Times, the US Food and Drug Administration (FDA) has been moving forward with several treatments and potential treatments for a variety of different…
Limb-Girdle Muscular Dystrophy or LGMD, is a form of Muscular Dystrophy caused by a mutation which disrupts production of the protein beta-sarcoglycan. There are currently no treatment options for LGMD and…
According to a story from tonic.vice.com, Liz Aronin and Jamie Mills both thought that everything was going smoothly with their newborn son Quinn, but within a few months, it became…
99% Invisible recently reported on the history of "Orphan Drugs." These drugs are an enormous source of income for pharmaceutical companies - but the explosion in their production is a…
Polycythemia Vera or PV, is a rare blood cancer. It's caused by a mutation which results in the overproduction of red blood cells. Too many blood cells significantly increases your…
According to a story from MarketWatch, Annie Wilson was diagnosed with spinal muscular atrophy when she was only six months old. With no real treatment options available, doctors told her…
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…
According to a story from prnewswire.com, the drug developer Mirum Pharmaceuticals recently announced that is has entered an agreement with Shire which grants exclusive rights for marketing and development of…
According to a story from ncbnewyork.com, a recent study found that $7 million on donations on crowdfunding sites for medical patients have gone towards treatments that are medically unsound and…
According to a story from Xconomy, the Rainwater Foundation, which was first founded in the 90s by Texas billionaire Richard Rainwater, is offering cash rewards to the tune of $250,000…
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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