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…
Maybe you’re someone like me who didn’t know what a Hemophilia Treatment Center is or what it does. I never thought about treatment centers of any type, of course, there…
Kids with spinal muscular atrophy (SMA) have a hard time with an evaluation method known as "pair-matching tests." They have a harder time identifying what number, letter, color, or object…
I first became aware of the bleeding disorder hemophilia when I was in high school reading about Nicholas II, the last Russian tsar. His son had severe hemophilia B, which…
When you’re a preteen, fitting in (or at least, not standing out) can be a big part of life. Sometimes, you’re just trying to get along with schoolmates, teachers, and…
You may have heard someone say: “You don’t look sick.” Few phrases can be as disheartening to someone battling a lifelong, or chronic, illness. Blood diseases are just one such…
The spinal cord is a major part of the central nervous system, and when it becomes inflamed, transverse myelitis results. It often causes a band-like sensation across the trunk of…
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…
Status epilepticus is a rare and sometimes fatal condition. About 60 percent of those with this super-refractory status epilepticus (SRSE) die, making it the most dangerous type of seizure. This…
Long have these cold-blooded, no-limbed reptiles bore a bad reputation; from the Bible to Disney to Indiana Jones. But are things about to change? According to a new French study…
Happy Independence Day Weekend Everyone! We have the latest update on the UK cystic fibrosis fight for Orkambi. We also have a great contribution on the importance of taking care…
According to the Salk Institute for Biological Studies, scientists have had a breakthrough in past efforts to develop a technique that can determine which proteins in a cell interact with…
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…
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…
Annually, 13 out of 100,000 people are diagnosed with trigeminal neuralgia. Trigeminal neuralgia is described as a sharp and intense pain that can occur in the face or mouth. The…
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…
Researchers wanted to know how likely it is for patients to have fibromyalgia with ankylosing spondylitis (AS). As a result, they found that particular comorbidity to be common. Marina Magrey,…
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…
A phase 3 clinical trial has produced positive results for cutaneous T-cell lymphoma (CTCL) patients. The drug tested is called brentuximab vedotin (Adcetris). Specifically, the drug was created to treat…
Case Western Reserve University researchers have recently developed gene-carrying nanoparticles that are able to bring healthy genes to those negatively affected by Leber congenital amaurosis (LCA), a disease that causes…
It's not unusual for a disease like psoriatic arthritis to come with the risk of having other conditions, or a comorbidity. More than you might think, people with psoriatic arthritis also…
Early this month, Novocure announced that the first patient was enrolled in the RTOG Foundation's Phase 2 pilot trial for recurrent glioblastoma. This trial is aimed to test the safety and…
According to an awesome story in DelRay Newspaper, Kelsey Diamantis had always dreamed of going to the Kentucky Derby at Churchill Downs to watch the horse race. While thinking about…
Biohaven Pharmaceutical Holding Company Ltd. announced at the end of last month that the company has completed enrollment in its Phase 3 clinical trial of trigriluzole in spinocerebellar ataxia (SCA)…
In April, Prometic Life Sciences Inc. announced it would be submitting a Biologics License Application to the FDA for treatment of hypoplasminogenemia. The company's therapy has already been listed with Orphan…
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