.
Just weeks after the FDA required more data prior to approving an oral form of Octreotide, proposed by Chiasma Pharmaceuticals, a British pharmaceutical and device company, Glide (not the dental…
Have a rare disease? Have you ever been to a national patient conference? These are invaluable opportunities to connect with others who share your challenges and concerns! Imagine being in…
“ Many describe the symptoms of HD as having ALS, Parkinson’s and Alzheimer’s – simultaneously.” - The Huntington’s Disease Society of America States For almost 50 years research into the cause and…
A mother of three in Scotland had experienced dystonia symptoms since childhood, and these symptoms got progressively worse over the years. Like so many with a rare condition it took…
The HAE community cheered in July of 2015 when another new therapy was approved by the FDA to treat acute HAE attacks. Now, the pharma company has another reason to cheer. The…
Life just got a little easier for parents of young kids with Tyrosinemia Type I (HT-1) Raising a child with Tyrosinemia requires constant, attention to diet, a special formula, and…
Wilson Disease: the rare, autosomal (meaning it occurs in both men and women), recessive (meaning that an individual must inherit one copy of the mutated allele from each parent to…
As families and individuals dealing with rare disease, we all want research and answers sooner- but we soon learn how slow that process is. But check out the hope below: 9500…
Mycosis Fungoides (MF) is a rare form of lymphoma that affects usually affects the skin causing inflammation, tumors, plaques, and redness. It only affects 3 to 4 people in a…
Dr. David Fajgenbaum was read his last rites- but he did not die, instead he turned the rare disease research model around. He is now working to expand the model…
Kids diagnosed with EB, or Epidermolysis Bullosa, have skin as fragile as a butterfly’s wings. DEBRA stands for Dystrophic Epidermolysis Bullosa Research Association, the patients association for this fragile skin disorder which…
According to PW writer Kathy, there was a shining star at Rare Disease Week on Capitol Hill's Rare Artist Reception. Olivia is the youngest of four daughters. She was diagnosed…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
