Study: PKU Patients Face a Higher Health-Economic Burden
Many rare disease patients are familiar with high prices for treatment. In fact, the most expensive medication in the world treats a rare disease: Zolgensa, a gene therapy for spinal…
Many rare disease patients are familiar with high prices for treatment. In fact, the most expensive medication in the world treats a rare disease: Zolgensa, a gene therapy for spinal…
Gene therapies are becoming an increasingly popular treatment for genetic diseases, especially rare ones. Now, medical professionals are trying a new strategy with gene therapy: adding acetaminophen, also known by…
The FDA approved Beovu, a treatment for wet age-related macular degeneration (wAMD), back in October of 2019. Now, not even two years later, there are changes that must be made…
Recent research conducted by Dr, Elif Oral has discovered that leptin therapy may be a viable treatment for nonalcoholic steatohepatitis (NASH), which is a form of fatty liver disease. Dr.…
The gut plays a big role in our bodies, we know that. While some of its roles are obvious, others are still being discovered. In fact, the microbiome of the…
Early diagnosis and prompt treatment are absolutely necessary for the best outcomes in many rare disorders. Keratoconus, a rare disease that impacts the cornea, is no exception. It is best…
It's no secret that COVID-19 can cause lingering symptoms, some of which persist for months. In fact, there is a name for those who deal with the lasting symptoms of…
Rare Bears started out as a small effort to support children with rare diseases. Now, it is a global non-profit that has an army behind it. This organization makes one-of-a-kind…
A recent study investigating treatment for adult acromegaly patients has revealed that switching from injectable somatostatin receptor ligands to oral octreotide leads to an improvement in quality of life and…
ACADIA Pharmaceuticals has released data on NUPLAZID, a selective serotonin inverse agonist and antagonist preferentially targeting 5-HT2A receptors. This treatment is meant to treat Parkinson's disease patients, specifically the delusions…
70-year-old Trevor Hardie from New South Wales is recovering after almost losing his life to leptospirosis, a bacterial infection that is spread through the urine of mice. He's not the…
Medical professionals are still trying to understand all of the underlying causes of Parkinson's disease, a progressive disease that sees the degeneration and death of motor neurons. A recent study…
Rare disease research is often lacking due to a lack of funding, interest, or awareness. To combat this, the Barth Syndrome Foundation (BSF) and American Heart Association (AHA) have joined…
Cure Rare Disease is a nonprofit organization that aims to help those who are impacted by rare diseases. Currently, the Boston-based company is planning a fundraiser to celebrate all of…
Diagnosis can be difficult to achieve for rare disease patients. Some patients experience years of searching for the correct diagnosis, with others receiving the wrong one along the way. Changes…
Pfizer Rare Disease is an approach taken by pharmaceutical giant Pfizer to develop treatments for rare and orphan diseases. In the past, the company has worked with the National Newspaper…
Brigatinib has been approved for the treatment of ALK+ metastatic non-small cell lung cancer (NSCLC), as well as ALK+ NSCLC patients who have received prior treatment with crizotinib. While it…
The diagnostic journey for rare disease patients can be long and frustrating. In fact, 28% of rare disease patients spent a minimum of seven years waiting for their diagnosis, and…
Intravenous immunoglobulin (IVIG) has been used to treat autoimmune conditions for years, and it is now being explored as a treatment for certain neurological diseases. Recent research has investigated IVIG…
Misophonia, which literally translates to hatred of sound, is a rare condition that causes affected individuals to have severe emotional reactions to common sounds, such as chewing or tapping. For…
Rare disease research faces many obstacles: lack of participants, lack of funding, and lack of interest are just a few. Now, the charity Cerebra has launched a network to take…
India has been dealing with a harsh second wave of COVID-19, resulting in 300,000 deaths as of Monday, May 24th. Now, there is a new fear for those who have…
Now that there are FDA-approved vaccines for COVID-19, millions are flocking to get their doses. A very small portion of these people are now experiencing myocarditis, and 18-year-old Evan Morud…
Molly Ringwald, the star of a number of classics from the 80's, is the spokesperson for the National Meningitis Foundation's new 16 Vaccines campaign. The campaign focuses on the second…
Feeling sore after an intense workout isn't out of the ordinary. It's normal for one's legs to feel a little wobbly or to hurt a bit while walking around. That's…