When it comes to researching and finding treatments for rare diseases, funding plays a crucial role. Recently, the Medical Research Future Fund (MRFF) Stem Cell Therapies Mission presented Monash…
There are multiple forms of Batten disease, each with their own distinct genetic cause. One of these forms is caused by a mutated CNL6 gene, but researchers were unsure as…
According to a story from The Boston Herald, a new treatment for acromegaly called Mycapssa was recently approved by the US Food and Drug Adminstration (FDA). Developed by Chiasma, a…
According to a story from BioSpace, the drug company Zynerba Pharmaceuticals Inc. recently released the results from a clinical trial. This trial was testing the company's product Zygel™ (CBD gel)…
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…
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…
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 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…
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…
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…
Acceleron Pharma has released the results from Phase 2 of their PULSAR trial, which studied sotatercept as a treatment for pulmonary arterial hypertension. Researchers are very excited by the positive…
uniQure has recently began their Phase I/II clinical trial of AMT-130, a gene therapy for the treatment of Huntington's disease. Two patients are included so far, with one being given…
Researchers believe that they may have found the cause of myasthenia gravis (MG), according to an article in Myasthenia Gravis News. A study found that issues with mitophagy in immune…
According to Parkinson's News Today, researchers have created a silicon-based antioxidant agent that can be supplemented into the diet to prevent neurodegeneration and issues with coordination and motor skills associated…
Pulmonary hypertension can be caused by a variety of other conditions, often making it difficult to distinguish. A problem that medical professionals often encounter is differentiating pulmonary arterial hypertension (PAH)…
According to Medical Xpress, a new gene therapy has been studied for the treatment of Duchenne muscular dystrophy. This treatment, SRP-9001, has been shown to deliver micro-dystrophin and improve functional…
This morning, SynAct Pharma AB announced in a press release that they began a Phase II clinical trial to determine the efficacy and safety of AP1189 on treating patients with…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
