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…
Patient Worthy is proud to announce we are participating in Patients as Partners EU in London, UK on January 23rd. Patients as Partners EU represents the only conference opportunity in Europe…
Nursing student Jess Boxley could be blind if she didn't see a doctor for her extreme headaches and neck pain, reported The Huffington Post UK. Jess, at a young 22-years-old,…
Margarita Matias could not believe her eyes when she walked into the newly renovated bathroom. Her old bathroom was far from ideal, and often leaked. The new arrangement is optimized…
According to a recent article from indiatoday, a new study has revealed information about how osteosarcoma and other cancer cells manage to survive in the body. The new research suggests…
According to an article from businesswire.com, the pharmaceutical company Odonate Therapeutics announced the beginning of CONTESSA, a phase three study of the treatment tesetaxel and its function in patients with…
Patients suffering from sickle cell disease, SCD, might just be getting news they've been waiting to hear for a long time. Just this week, the U.S. Food and Drug Administration…
At three months old, Emily's mouth hurt. Her first tooth started coming in. Her parents immediately knew that something was wrong. Three months later, the family received dire news about…
While it's general knowledge that working out is a huge plus for a healthy life, many questioned whether it was positive or dangerous to individuals with rare diseases. Yet, BMC…
According to a story from philly.com, many people with severe forms of brain cancer are not receiving sufficient hospice treatment, according to a recent assessment. The study took data from…
According to a story from financialbuzz.com, the pharmaceutical company AveXis, Inc., is planning to begin preparations to submit a Biologics License Application (BLA) to the Food and Drug Administration for…
According to a story from the Chicago Tribune, Ben Barres, a widely renowned neurobiologist and researcher, passed away at age sixty-three after fighting pancreatic cancer. Mr. Barres was credited with…
5-month-old baby, Rohaan's, life was spared when he recently underwent surgery to treat his rare congenital heart condition, surgery that almost didn't happen, reported Times Of India. His parents, Muhammad…
Watch Prof. Charles Craddock discuss important and heartening results he and his team presented at the American Society of Hematology 2017 Annual Meeting and Exposition in Atlanta, GA - regarding whether there…
When you are in the midst of difficult times in your life, what do you do to make it through? Do you try and find a way to hide out…
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