PDUFA Fee Waived for Gastroparesis Treatment
According to a story from Financial Buzz, the pharmaceutical company Evoke Pharma announced that the U.S. Food and Drug Administration has approved a small business waiver for Gimoti, a nasally…
According to a story from Financial Buzz, the pharmaceutical company Evoke Pharma announced that the U.S. Food and Drug Administration has approved a small business waiver for Gimoti, a nasally…
Ellie White is everything you would want in an 18-month-old daughter, fun, playful, and full of life-- yet not too long ago her family discovered she had a rare disease,…
Just yesterday, the incredibly successful actor Irrfan Khan, announced on twitter that he just found out he has a rare disease, reported The Guardian. Khan has appeared in over 100…
Happy Friday, Patient Worthians! Today, we're highlighting a story about the donors who sacrifice their kidneys for rare patients, an update on CF research, and an exciting orphan drug designation.…
The day after Christmas in 2016 brought a post-Holiday surprise to many in the U.S. with spinal muscular atrophy. December 26, 2016 marked the day that Spinraza was approved by…
According to an article from kait8.com, the pharmaceutical company Yisheng Biopharma announced that YS-ON-001, a leading candidate for the company, received Orphan Drug Designation from the U.S. Food and Drug…
According to an article from lymedisease.org, Dr. Raphael Stricker is slated to speak about the future of Lyme disease diagnostics at MyLymeData2018: Seeking Cures Together. This conference is intended primarily…
UCLA conducted a new study that looked at patients with claustrophobia and attempted a shortened MRA scan. They hoped to obtain the same quality of images that the current, longer…
Haley Hayes is a normal pre-teen, yet she suffers from Pompe disease. Pompe disease is a genetic condition, which causes muscle and organ impairment. Early diagnosis and enzyme replacement therapy…
According to an article from PRNewswire, recent research may have discovered why glioblastoma tumors develop resistance to treatment. Glioblastoma is a deadly form of brain cancer that is known for…
According to a story from bioportfolio.com, The California Institute for Regenerative Medicine has given $5.74 million to the City of Hope, a private, nonprofit clinical research center that is primarily…
Life for 27-year-old Medical Bio Science graduate, Tate Nova Scanlon, has never been easy. As originally reported by The Lincolnite, Tate has been battling aggressive symptoms caused by adrenal…
According to a story from unmultimedia.org, a recent report has revealed that there are twenty countries across the world that still discriminate against people with Hansen's disease, also known as…
According to a story from Politico, the two legislative bodies of the Virginia General Assembly, the House of Delegates and the Senate of Virginia, passed vastly different budgets last week,…
Brandi Lawrey describes her son, Gavin, as a little man because he continually shows bravery and courage well above his age, reports News-Press. Gavin was diagnosed with a rare mitochondrial…
Fashion blogger Yavanna Keogh who runs Makeupmonster just took to social media the devastating news of her son's recent diagnosis, first reported by Mirror. Yavanna and her partner from Dublin,…
Happy Rare Disease Week, Patient Worthians! As we wind up after a week of celebrating the rare patient community, support networks, and advocacy, we want to highlight four rare disease…
Corbus Pharmaceuticals has received the Development Award presented by the Cystic Fibrosis Foundation, along with $25 million to support an upcoming study, reports Financial Buzz. Corbus plans to test out…
According to an article from Khon2, locals on Honolulu Island in Hawaii are starting to raise concerns after the children of three firefighters from the Waipahu Fire Station were diagnosed…
Dena Winchester, a sophomore at Rutgers University, faces the struggles that come with her condition by advocating for change, reports New Jersey Jewish News. After she was diagnosed with lipodystrophy…
According to a story from ABC News, medical researchers are beginning to monitor the long term health of people that have donated one of their kidneys. A kidney transplant can…
According to a story from the National Organization for Rare Disorders, patient advocacy organizations are joining forces to send letters to Medicaid directors across the USA to advocate for the…
Alondra, a 14-year-old freshman in high school weighs only 65 pounds. This is a seemingly easy giveaway that she was sick, but initially the weight loss was attributed to her…
According to a story from BBC, a former employer is paying for a woman's cancer treatment. 74 year old Pamela Stubberfield of Oxfordshire was diagnosed with mesothelioma. More than 80…
Jahkee Johnson, a 16-year-old from Cedar Grove High School, is exceptional and accomplished for many reasons, beyond the fact that he tackles challenges and goal without the help of legs,…