Potentially good news arrived for patients with hereditary TTR amyloidosis, as the treatment drug inotersen received it's New Drug Application approval and will go into review by the U.S. Food…
19-year-old Jonathan Inkin created a "20-strong" bucket list with intention to cross them all off before his birthday. Thanks to the help of celebrity Toff (Georgia Toffolo), the winner of…
Tiffany and Caleb isn't your typical engaged couple. They are passionately in love and excited for their wedding, but their romance could literally end any moment. NBC 5 Chicago reported…
Reported by Fox 2, Anthony Anzell doesn’t let his rare genetic disease hold him back. Anthony is an extreme athlete who just swam 19 miles, the entire length of Torch…
The Hempel family decided to take action against Vtesse and Sucampo Phamaceuticals who allegedly stole and sold their private information for profit, reported Forbes. The Hempel family has two twin…
Even after the third denial from the U.S. FDA, Food and Drug Administration, PTC Therapeutics Incorporation will not stop fighting for the drug Translarna that they believe will help patients…
The Wahlstrom family of four, is getting a miraculous gift to help care for their two children suffering from Batten disease, reported CBS Minnesota. With the love of their local…
The FDA, US Food and Drug Administration, unanimously agreed upon the recommendation for a new gene therapy that treats a rare form of blindness, reported ScienceNordic. Spark Therapeutics, a biotech…
If you live near Pompano Beach in Florida, the Water Balloon Bulls-eye charity event is not to be missed, reported Sun Sentinel. The event is focused on raising money, as…
CISCRP, The Center For Information & Study On Clinical Research Participation, is a unique and one-of-a-kind non-profit focused on clinical trial education. CISCRP is wants to bring the need for…
Blessings amongst the hardest time in Jessica Boesmiller's life came in the form on twins, reported News.am. 37-year-old Jessica is a mother of four and recently learned in November that…
The Mastocytosis Society is an amazing organization that brings focus to patients with mastocytosis and mast cell activation diseases. They hope to bring awareness, as well as education to patients,…
Ellie Simmonds is a dominate Paralympic swimmer having won five times in her 23-years and was honored locally for her success, reported Border Telegraph. Just recently, she attended to a…
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…
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…
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…
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,…
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…
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…
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,…
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…
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…
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…
Nikki Oosthuizen gave her 16-year-old daughter, Kim, a kidney as she suffers from a rare kidney disease, yet only four days later the disease returned and destroyed her kidney function…
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