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.…
George Mendoza has inspired others throughout his whole life. He has been an athlete, an author, a motivational speaker, and more. Born in 1955 in New York City, Mendoza has…
A recent study published in Arthritis Care & Research illustrated how higher dosages of prednisone lead to better outcomes for lupus nephritis (LN) patients. After one year, participants were found to…
Back in October, the International Pemphigus & Pemphigoid Foundation (IPPF) took another step in their plan to learn from and work with patients to increase awareness for pemphigus. They held…
Chronic kidney disease (CKD) affects hundreds of millions of people throughout the world. As of 2017, 697.5 million people were estimated to have CKD by this study published in Nature Reviews…
bluebird bio has created a gene therapy for the treatment of cerebral adrenoleukodystrophy (CALD), which is now making its way through a clinical development program. According to Businesswire, bluebird has…
A new study is in the works, and it will investigate how cystinosis, a genetic disorder characterized by an accumulation of the amino acid cystine in the cells, leads to…
Newron Pharmaceuticals and Zambon have entered into an agreement that has the potential to produce a label extension for a treatment for Parkinson's. The agreement is focused on safinamide as…
Pamrevlumab is a first-in-class antibody that treats Duchenne muscular dystrophy (DMD) by inhibiting the effects and activity of connective tissue disorder growth factor (CTGF). It is being developed by FibroGen,…
According to Charcot-Marie-Tooth News, the CMT Research Foundation has announced that they will provide additional funding for the development of a gene therapy for Charcot-Marie-Tooth disease Type 1 (CMT1A). The…
The French National Agency for Medicines and Health Products Safety, also known as ANSM, has granted Temporary Authorization of Use of berotralstat for those living with hereditary angioedema (HAE) above…
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