While most of us have trouble accepting our limitations, it can be especially hard for people whose limitations are due to a rare disease. In meeting with and corresponding with…
"Do you know what the world’s third most populated country is?" It's a question that Dr. Rafael Sousa Fernandes posed in an OpEd, wrote that made all of us here at Patient…
Rare disease research is often underrepresented in the field of science. However, recent developments have made researchers more interested in studying this group of diseases. For instance, investments in gene…
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…
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 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…
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…
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…
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…
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 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…
Neuroendocrine tumors are growths which form from neuroendocrine cells in various places throughout the body. They can occur in the gastrointestinal track, the pancreas, the lungs, the thymus, and other…
Biobanks store biological samples to help scientists research certain diseases. Registries collect information from patients and standardize it, but no samples are stored. Which of these is best for rare…
Batten disease is a lysosomal storage disorder. It's neurodegenerative, meaning it results in the deterioration of neurons. One of the most common forms of this disease is caused by mutations in the…
According to a story from University at Buffalo, a recent discovery of a new genetic mutation continues to build on decades of research from Dr. Richard Gronostajski, a professor and biochemist…
According to a story from the Australian Jewish News, a Jewish teen named Gidon Goodman was born with Gaucher disease. Recently, he started the first Gaucher disease patient support group…
According to a story from Raconteur, finding sufficient funding for the development of new therapies for rare diseases has always been a major challenge. Rare diseases are defined in the…
According to a story from nature.com, Amber Sapp's twelve year old son Garrett had been participating in a clinical trial that was testing a new potential treatment for his Duchenne…
According to a story from UCI News, University of California Irvine scientists have developed an innovative immunotherapy that could screen patients in order to analyze the composition of their tumors…
According to a story from EurekAlert!, researchers have been scrambling to find a useful treatment for acute flaccid myelitis, a rare disease that has been appearing in unusual numbers in…
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