According to a story from pharmavoice.com, the pharmaceutical company Cadent Therapeutics has announced the beginning of the Phase I clinical study for its drug candidate CAD-1883. The therapy is a…
According to a story from EurekAlert!, scientists from UCLA have developed a new method of delivery for gene therapies called nanospears. These structures could allow for more accurate and precise…
Much has been said about the way in which social media is affecting society today. Recently, many of the remarks have been negative. Yes, it has been used by some…
According to a story from raredr.com, The U.S. Food and Drug Administration (FDA) has recently approved Hizentra as a maintenance therapy for patients with chronic inflammatory demyelinating polyneuropathy. Hizentra is…
According to a story from raredr.com, the FDA had to order the cessation of Phase II/III trials for the microdystrophin transfer therapy SGT-001, which was in development for the treatment…
A “Right to Try” bill is currently being discussed by Congress. Advocates on both sides of the story present valid points to either pass or stop the bill. That’s why…
In a previous article we discussed what the Right to Try bill is, and what it’s current status was in the US Congress. It recently failed to pass in the…
A team lead by geneticist Christopher Gregg from the University of Utah conducted a study on elephant genes that discovered why elephants don't get cancer, reported by News 4 Tucson.…
Happy St. Paddy's everybody! While most people are putting on their green clothing to celebrate the holiday, some communities of rare patients have other exciting happenings in mind. This week,…
Novartis and Pear Therapeutics have joined forces to develop digital therapeutic software applications to treat patients with schizophrenia and multiple sclerosis, reports World Pharma News. Novartis is known for the…
According to a story from Charcot-Marie-Tooth Disease News, the U.S. Food and Drug Administration (FDA), under the leadership of commissioner Scott Gottlieb, has begun to change clinical trials by accepting…
According to a story from wired.com, Stephen Hawking, one of the most famous physicists on the planet, passed away on Wednesday, March 14th, 2018. He was 76 years old. His…
According to a story from All4Women, Nicole Capper, crowned Mrs. South Africa, is planning to summit Mt. Everest, the highest mountain peak on the planet, in order to help promote…
Forty-eight-year-old Isabel lives with the genital skin condition called lichen sclerosus, being diagnosed post menopause, and it's not an easy new life-style, reported NetDoctor. Lichen sclerosus is a rare genital…
The Southern Daily Echo originally reported that a majority of pancreatic cancer patients don't receive life-extending treatment after diagnosis. Recently, the Pancreatic Cancer UK charity reported that only a small…
A Boston biotech company called FNDA has collaborated with research organizations to utilize artificial intelligence, AI, to aid drug development and disease diagnosis, reports FierceBiotech. They are currently looking for…
According to a story from the Wall Street Journal, the U.S. healthcare system might be able to learn a thing or two from Netflix. A well understood aspect of healthcare…
According to a story from PressReleasePoint, a new partnership has developed to generate artificial intelligence technology that will help diagnose people with a variety of rare diseases. The partnership consists…
According to a story from fiercebiotech.com, the gene therapy startup Generation Bio managed to fundraise $100 million in a little over a month. In its prior series A round of…
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…
Macey Brietenback continues to defy all odds. Originally reported by CBS Baltimore, the teenager lives with a condition that prevents her from digesting food. Even though it has given her…
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.…
Making big changes to your eating plan can present a challenge for some. Now, factor in having hemochromatosis—a rare condition characterized by the buildup of iron—and you’ve got the ingredients…
Many runners shared their inspirations after the Little Rock marathon this week. Among them Nancy Gifford, a thyroid cancer survivor, finished the race. Keep reading to learn more about her…
33 rare disease organizations across the country have teamed up for $1 million fundraising goal during the months of February and March to fund research and support patients, in honor…
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