Midatech Pharma just received verbal confirmation from Polish regulators to conduct the first European in-human study for a treatment that could potential aid those with carcinoid cancer and acromegaly, reported…
According to a story from CBC News, Jillian Lanthier's son Landen Alexa was diagnosed with a severe and rare form of juvenile idiopathic arthritis at six years old. Although he…
Carl Luepker was diagnosed with dystonia when he was 12-years-old, reported KALW. The twitching started when he was 10-years-old. This quickly started affecting his piano playing, something he loved so…
According to a story from Syosset Jericho Tribune, the charity basketball fundraiser called Hoops for Harrison is back again this year. The tournament was named after Harrison Gillman, who now…
According to a story from ANCA Vasculitis News, The European Medicines Agency just approved the pharmaceutical company ChemoCentryx's application for the marketing of avacopan as a potential treatment for anti-neutrophil…
Although it never should have happened, at least the right course of action was taken! Reggie Bibbs of Houston, Texas was turned away from his Uber driver when he requested…
One of Patient Worthy's partners, The MDS Foundation, will be hosting another FREE event! When: February 3rd, 2018, 9:30am – 2:00pm Where: San Diego Marriott La Jolla Hotel Soledad Ballroom…
REGENXBIO announced last month that the Investigational New Drug application (IND) is active for a Phase I/II clinical trial of RGX-121 for the treatment of Mucopolysaccharidosis Type II (MPS II)…
At only six years old, Simeon Augustus Peterson, also referred to as "Mr. Pete," was diagnosed with Hansen's disease. From then on, he lived the rest of his life cut…
January 5th was not only a great day for the Butler basketball community, it was a dream come true for the bulldog fan Seth Dennison, reported Fox 59. The 7-year-old…
According to a story at medpagetoday.com, a recent study showed that stem cell transplant could be a viable new method for treating scleroderma, and could be more effective than previous…
According to an article from Financial Buzz, the pharmaceutical company CymaBay Therapeutics has announced that the first of its patients have begun to enroll in a long term seladelpar clinical…
RespireRx Pharmaceuticals announced last month it was cleared to resume its ampakine CX717 clinical trials to evaluate the drug’s efficacy in several central nervous system and respiratory disorders, including Pompe…
Shaquem Griffin, an inspiration on and off the field, recently received the Rare Disease Champion Award presented to an outstanding leader in college football and the rare disease community alike,…
Last month, the FDA has granted Achillion Pharmaceuticals orphan drug designation to ACH-4471 for the treatment of patients with C3 Glomerulopathy (C3G). According to the FDA, the Orphan Drug Designation program provides…
New research is helping experts understand the underlying causes of idiopathic pulmonary fibrosis on the molecular scale. When solving any problem, disease included, it is best to start by understanding…
Nothing is worse for a family than hearing the word “cancer,” especially when it involves a child. The Tyson family was ready to start their perfect life eleven years ago.…
New research is being conducted to develop new technologies to detect ovarian cancer. Keep reading or follow the original story here to learn more about the struggles and developments involved…
Madison Simons, a 20-month-old child has visited the hospital more than 12 times and spent more than 50 days at the Baylor Scott & White McLane Children's Hospital this passed…
According to a story from the National Hemophilia Foundation, the Swedish pharmaceutical company Orphan Biovitrum announced that its first patient was registered for a new phase 4 clinical trial. The…
Another successful partnership in the rare disease community has come to fruition, reports The Pharma Letter. Just this past week, CureVac and Arcturus Therapeutics will become a powerhouse together with…
There are many reasons to celebrate the life of genius physicist Stephen Hawking, but celebrating his 76th birthday is definitely extra special, reports Rare DR. As many know, Hawking suffers…
Happy Friday, Patient Worthians! This week we have articles about people working together on different types of rare disease teams. We have stories from a family who supports two children…
Is thoracic outlet syndrome ending careers in the NHL? It’s certainly more common athletes. Keep reading or follow the story at USA Today to learn more. The New York Rangers…
A new study uncovered two proteins that may be able to reduce symptoms in certain diseases. The proteins may be especially effective at treating diseases with nonsense mutations, such as…
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