β-thalassemia major is a rare blood condition that necessitates a lifetime of blood transfusions. Despite advancements in treatments, the perfect cure has yet to be found. Bluebird Bio, Inc. is…
No two patients a doctor sees will ever be exactly alike. They all will have different symptoms, allergies, and conditions, in addition to different backgrounds, personalities, and identities. While you…
According to Port City Daily, 21-year-old Haley Swanson has been living with dermatomyositis for 10 years. She was diagnosed at age 11 with the autoimmune condition, which means that her…
According to Today, Quinn Hoover is an 8-year-old boy who lives in Nebraska. His body doesn’t produce enough alkaline phosphatase enzyme, causing his bones to be extremely “soft." The condition…
Carol Littrell and Cindy Hamilton were coworkers. And according to JDNews, Neither imagined they would ever become much closer than that. But everything changed by one chance encounter. Carol has…
An Indian family found out their little girl, Parvathy, only three months old, had primary hyperoxaluria type 1. In simple terms, those words meant she was born without the enzyme…
Imagine living every day with fear that paralysis may be coming. The idea always lingering in the background, ominous. That's the case for Kam Redlawsk. But she does not let…
According to the Daily Mail, Harley Bond is a 4-year-old living in the UK. He has MPS III, or Sanfilippo Syndrome, a rare disease which has no treatment and no…
Kelley Fox is 23 years old, but developmentally she’s only around 5 or 6 years old. She lost half of her brain due to a disorder called tuberous sclerosis. The…
Not only does Pamela Looney have hypereosinophilic syndrome (HES), she also has asthma and has suffered from a tumor. At only 15, the sudden development of this tumor severely affected…
Rare disease research usually isn’t the first thing med students think to specialize in. Mostly, because it’s such a small field and it’s not extensively covered during medical school. Let's…
In June 2017, a film was released showcasing the story of a young girl named Kennedy Hansen who had Batten disease—a disease that causes a damaging buildup of lipofuscins in…
Feeling alone in your struggles is almost an inevitable side effect of living with a rare disease. You’re unlikely to have a support group in a very close vicinity and…
Allergan and Paratek Pharmaceuticals Inc. recently tested a drug called sarecycline for treatment of severe acne. The medicine is a tetracycline-derived antibiotic that is taken orally, and the best part…
According to QC Online, at six months old, Lorelei Lovelady was diagnosed with primary hyperoxaluria type 1 (PH1). Now, almost 9 months, the disease has caused her to need a new liver…
The Annual European Congress of Rheumatology (EULAR) press conference showcased two very promising treatment options for psoriatic arthritis (PsA). Both treatments show promise in limiting the inflammation associated with the…
Jessie Parks is a writer for the Charleston City Paper. Her words are absolutely beautiful to behold. The reason she started her column? Her mother. who was diagnosed with Waldenstrom macroglobulinemia…
In the US, the rare condition glomerulonephritis is the third leading cause of the last stage of chronic kidney disease (CKD). A clinical trial sponsored by the National Institutes of…
Contagion Live (Infectious Diseases Today) recently discussed a journal article on the prevalence of atopic dermatitis. The simple summary? More research is needed to uncover just how common this condition is. BUT…
During this year’s International Workshop on chronic lymphocytic leukemia (CLL), Shih-Shih Chen, PhD, was recognized for her innovative research. Her research could mean a cure is around the corner for CLL.…
By examining a zebrafish, scientists have found the gene that affects the maintenance of cone photoreceptors and visual functions in the eye. It might be the knowledge that’s been needed…
Too often rare diseases such as tardive dyskinesia (TD) are treated with medications and therapies that were made for other illnesses. This occurs pre-diagnosis, by physicians who have never seen…
According to the Daily Mail, Clare Fray has alpha-1 antitrypsin deficiency. But for years she thought her symptoms were just a part of normal life. She suffered from numerous chest…
It’s amazing how much we take for granted. Eating. Seeing. Walking. There are a million and one things that can go wrong with the human body, and until one of…
8-year-old Carter Anderson has PKAN. He’s only had his official diagnosis for about a year, as his parents were first told he had cerebral palsy. Since Pantothenate Kinase-Associated Neurodegeneration (PKAN)…
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