A recent study published in Frontiers in Immunology investigates the immune response to bone infections, such as Staphylococcus aureus, that cause osteomyelitis. Their research stands to give medical professionals a better…
A study published in Physician's Weekly investigated the visual outcomes, clinical features, and treatment of individuals with occlusive retinal vasculitis (ORV), a condition that is characterized by inflammation in the vessels of…
One of six-year-old Jackson Garwood's favorite things to do is listen to his father sing. Some of his favorite numbers include 'Talk Tonight' by Oasis and Aqualung's 'Brighter Than The…
Three people in the state of Indiana live with Niemann-Pick disease type C (NPC), and Audrey Mischler is one of them according to WTHI-TV 10. She was diagnosed very recently,…
The National Institute of Health (NIH) has created a new approach to diagnosing and treating methylmalonic acidemia (MMA), a rare genetic disorder that stops the body from metabolizing fats and…
Timothy and Andrew Revell are two brothers born with Duchenne muscular dystrophy (DMD), a rare neuromuscular disorder that sees the progressive wasting and weakness of the muscles. This condition has…
Arimoclomol is a treatment that is in development for ALS, Niemann-Pick disease type C (NPC), and inclusion body myositis (IBM). A recent Phase 2/3 trial has ended in failure, unable…
The FDA has recently provided 89bio with guidance for their upcoming clinical trial of BIO89-100, a treatment for nonalcoholic steatohepatitis (NASH). The trial will advance as planned, beginning in the…
Steven Elvidge has been left confused, frustrated, and debilitated for the past three years due to a mystery illness. Various symptoms have left him unable to live independently, forcing him…
Rare disease patients throughout India have expressed their disappointment and anger towards the country's new policy centered on rare conditions. One of their main concerns is that the policy changes…
The European Commission (EC) has granted the Orphan designation to Passage Bio for their Krabbe disease treatment, PBKR03. This adds another designation to the medication's list, as it has already…
Connor Dobyn's parents recognized that their son was missing important developmental delays early on. They brought their concern to the doctor, where Connor was diagnosed with autism. For the next…
On April 2nd, NFL scouts made their way to Eugene, Oregon to check out some of college football's most talented players. They watched as athletes completed the vertical jump, 40-yard…
Orphazyme's latest trial has ended in failure after arimoclomol, a possible treatment of inclusion body myositis (IBM), failed to meet any of its endpoints. As the drug has shown massive…
According to Yahoo Finance, D&D Pharmatech has initiated dosing in their Phase 1 trial of DD01, a treatment for nonalcoholic fatty liver disease (NAFLD) and diabetes in obese patients. As…
Spinal muscular atrophy (SMA) patients aged two months and older will now have an at-home treatment option in Europe. The European Commission (EC) has approved of Evrysdi for those who…
Recent research indicates that microRNAs may be a potential biomarker to diagnose and judge a Parkinson's patient's prognosis. A study titled “Deep sequencing of sncRNAs reveals hallmarks and regulatory modules…
Fatty liver disease, which can be caused by alcohol consumption or can be unrelated (nonalcoholic fatty liver disease/NAFLD), belongs to a larger group of conditions that have been labeled as…
Many rare disease patients face difficulty when it comes to diagnosis. In fact, it takes an average of five to seven years for them to finally receive the correct diagnosis.…
Caroline is the adorable three-year-old daughter of Kevin and Kelly Brennan Culver, and she was diagnosed with juvenile dermatomyositis (JDM) about a year ago. This means that she requires an…
Leigh syndrome is a rare mitochondrial disease that is not yet well understood by medical professionals. When this lack of comprehension exists, it is impossible to create treatments or improve…
Rare disease patients face a number of obstacles when it comes to getting the proper diagnosis and treatment. There's a lot of work being done to address these problems, some…
Tanner McLeod has been searching for a stem cell donor for a long time. He was diagnosed with sideroblastic anemia at just six months old and has required a transplant…
Recent results of a Turkish trial have demonstrated the need for further research into IFN-gamma as a treatment for Friedreich's ataxia (FA). This study showed that the therapy resulted in…
MSN News recently interviewed Dr. Laxmikant Palo, with the conversation focusing on the situations of people living with rare diseases in India and how to improve them. According to Dr.…
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