At the end of June, the National Hemophilia Foundation (NHF) released a warning to patients with hemophilia regarding AT132, an investigational gene therapy candidate. Developed by Audentes Therapeutics, AT132 uses…
If you're experiencing a difficult time with COVID-19, you're not alone. Many people, including myself, are struggling with managing anxiety, stress, and isolation. Additionally, this time period can feel traumatic,…
Vision loss is a major symptom of Stargardt disease, so a main goal of treatments is to combat this loss. In order to test the effectiveness of these treatments, there…
Medical professionals have found that the melatonin levels of Parkinson's patients impact their symptoms, specifically the gastrointestinal, sleep disturbance, and cardiac symptoms. Parkinson's News Today reported on a study that…
Recurrent Pericarditis Pericarditis is a rare autoinflammatory cardiovascular condition. It can lead to changes of the electrical conduction of the heart and a buildup of fluid surrounding the heart. Pericarditis…
Celltrion Healthcare has just announced positive progress for extending the authorization for the subcutaneous (SC) form of Remsima to five more autoimmune conditions. These are- Psoriatic arthritis Ankylosing spondylitis (AS)…
Scientists and researchers have long known how important glial cells are to the nervous system. Not only do these cells make up a majority of cells within the central nervous…
A new study published by ACS Nano and conducted by scientists at the University of Illinois at Urbana-Champaign uncovered a new way to modify stem cells. This study was conducted…
My name is Mika Jayne Covington. I’m 29 years old and I live with an ultra-rare disease called cystinosis. I am also a transgender woman and my pronouns are she,…
We are learning new things about COVID-19 every day. One of those things is the side effects, both neurological and physical. In fact, researchers from the UK have found that…
Researchers at Seelos Therapeutics are planning an animal study to evaluate SLS-007, a treatment for Parkinson's disease. According to an article from Parkinson's News Today, this study is possible after…
Generating neurons is only one of many goals that have so far escaped scientists worldwide. According to an article in Yahoo, the lead author of a new study in the…
CytRx Corporation has released the results of the second phase of their study of arimoclomol as a treatment for Gaucher disease. The data was positive, and it demonstrated this therapy's…
According to a recent press release, biotechnology company Cancer Prevention Pharmaceuticals (CPP) submit a New Drug Application (NDA) for accelerated approval of their drug therapy CPP-1X/sul to treat familial adenomatous…
Happy Thursday! This edition of the Patient Worthy Editor's Choice is coming to you a little early this week. If you're reading from the United States, we hope you enjoy…
Without treatment, spinal muscular atrophy (SMA) is the top cause of infant or child mortality before age 2. As a result, early detection is crucial in improving patient outcomes. But…
According to a recent article in Parkinson’s News Today, researchers at the UCSF Weill Institute discovered that disturbances in circadian rhythm increased a person’s risk of Parkinson's disease threefold. Circadian…
Scientists from the Universities of Edinburgh and Cambridge in collaboration with a research center in Germany have analyzed a DNA-damaging chemical that has given scientists a better understanding of the…
Sound Pharmaceuticals announced the first-ever Phase III clinical trial protocol for Meniere’s disease to be approved by the FDA. Meniere’s disease (MD) is a neurotologic disorder that involves tinnitus (ringing…
A study in the Journal of Movement Disorders on patients with Parkinson's disease has found a relationship between sedentary living and attention difficulty. Exercise is the age old medicine, with…
As shared in Medscape, the FDA recently approved oral octreotide (Mycapssa) for the treatment of patients with acromegaly. These delayed-release capsules are the first approved oral somatostatin analog ever approved.…
60% of all those diagnosed with multiple sclerosis (MS) also suffer from some form of sleep disorder. Fatigue is a more common symptom than even the well-known tingling and numbness…
Macular disease or stargardt disease, is a rare condition whose onset can occur at anytime. It results in damage to the retina, though most patients do not become completely blind.…
A recent study has demonstrated that through standardizing the whole genome sequencing process and making it routine for patients, we can improve rare disease diagnosis across the board. Earlier diagnosis…
Genentech has recently released the data from its SUNFISH trial, a study of the effects of risdiplam on spinal muscular atrophy (SMA) patients. Preliminary data from the JEWELFISH trial has…
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