According to a story from The Straits Times, Nicole Lim, who recently turned eighteen, learned that she had three different rare diseases when she was just thirteen years old. Getting…
Myasthenia Gravis (MG) is a progressive autoimmune disorder which results in the weakening of muscles. Most people with the condition are able to manage their symptoms with treatment and have…
According to a story from drugs.com, the biopharmaceutical company Catabasis pharmaceuticals recently provided updates on the status of its Phase 3 clinical trial testing its investigational therapy CAT-1004, also known…
According to the Boston Globe, the president of the Massachusetts Biotechnology Council has a personal stake in his continued advocacy for pharmaceutical development in his home state. Robert Coughlin has…
A new bronchiectasis research study was added to the clinicaltrials.gov website on New Years Day. This observational study, called the BRIDGE Study (Bronchiectasis Research Involving Databases, Genomics, and Endotyping) is…
EndoLogic’s study of their new gastroparesis drug, renzapride, showed no evidence of cardiotoxicity. Renzapride is a 5-HT4 agonist and 5-HT3 antagonist that has been successful in treating the symptoms of…
Yesterday marked the beginning of season 23 of The Bachelor on ABC-- a TV show that I am told I invest in to an embarrassing degree, and yet, somehow, feel…
When you're told your child has a rare, life-threatening condition you exhaust every resource trying to find answers. But the rarer the condition, the harder those answers are to find…
According to a story from globenewswire.com, the messenger RNA therapeutics company Translate Bio recently released the latest updates in regards to two of its leading product candidates for the treatment…
An article at ChrisD.ca recently shed a light on the difficulties of dating and intimacy when living with a terminal condition. Intimacy can be a stressful thing under the very…
Originally reported in a story from CNN, two year old Zainab Mughal from Pakistan has been fighting neuroblastoma- and in order to survive her treatment, she is going to need blood…
According to a story from EurekAlert!, the results of a recent Phase 3 clinical trial suggest that the targeted therapy sorafenib could be an effective treatment option for desmoid tumors.…
The Kids Wish Network (KWN) works to provide positive experiences for children age 3 to 18 who are living with life-threatening conditions. With the help of various sponsors, they are…
According to a story from WebMD, the US Food and Drug Administration recently issued a warning about a class of antibiotic drugs called fluoroquinolones. They have been linked to an…
Batten Disease There are 13 different forms of Batten Disease defined by the medical community. Each presents similarly but there are differences in the severity of symptoms as well as the…
"I feel powerless to help them. There needs to be a change. And change ought to begin in the form of a policy shift." These are Jay Avasarala's words in…
According to a story from Gizmodo, the genetic mutation which causes certain types of dog breeds, such as Boston terriers and bulldogs, to have spiral shaped, corkscrew tails appears to…
According to an article at PR Newswire, Bridge Biotherapeutics Inc., a South Korean biotechnology company, has announced the opening of three of its scheduled ten clinical study sites for the…
According to a story by MarketScreener, pharmaceutical company Acer Therapeutics Inc. has submitted a New Drug Application (NDA) for its vascular Ehlers-Danlos syndrome (vEDS) drug Edsivo. The NDA was granted…
Neuromyelitis optica (NMO), or Devic's disease is a rare autoimmune condition which causes inflammation in the optic nerve and/or spinal cord. The condition affects 5 in every 1,000,000 people and…
Limb Girdle Muscular Dystrophy Type 2D (LGMD2D), also called alpha-sarcoglycanopathy, is a form of muscular dystrophy which is caused by a defected SGCA gene. It causes the body to lose function of…
CRISPR Therapeutics and Vertex Pharmaceuticals have been collaborating to develop a new treatment option for Sickle Cell Disease. It's called CTX001, a type of stem cell therapy. This drug has…
Thanks to a Phase 2 trial, pediatric patients with Philadelphia chromosome-positive acute lymphoblastic leukemia or Ph+ ALL, now have a new treatment option. The FDA has announced approval of Sprycel…
It has just been announced that Capricor Therapeutics has put their Duchenne Muscular Dystrophy (DMD) clinical trial on hold. Fortunately, the company does plan on continuing the trial. What is…
According to a story from ESPN, there were times when Lynn Rogers wasn't sure if she was going to be able walk, much less run, ever again, but she wound…
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