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…
Botox Botox was first approved as a treatment for strabismus and blepharospasm, rare eye conditions, in 1989. Now, it is approved for 11 different conditions including cervical dystonia, chronic migraines,…
By engineering exosomes, Sarepta Therapeutics believes they can utilize gene therapies, RNA therapies, and gene editing without triggering an immune response in the body. They have just announced a new…
Ultragenyx Pharmaceutical's therapy, CRYSVITA, has recently been approved by the FDA for the treatment of FGF23-related hypophosphatemia in tumor-induced osteomalacia (TIO) associated with phosphaturic mesenchymal tumors. This approval comes after…
According to a story from PR Newswire, the US Food and Drug Administration (FDA) has recently approved a new treatment for Dravet syndrome, a rare disorder that causes seizures and…
RARE on the Road: a Rare Disease Leadership Tour June 27th, 2020 New Orleans, LA The Rare Disease Leadership Tour is designed to give insights and education to rare disease…
For most drug therapies, researchers test on animal models of a disease before testing on humans. Recently, using microRNA-483, a small RNA molecule, on rat models of PAH reduced…
As the medical field advances, so do drug therapies and treatment options. According to Asbestos.com, which is headed by The Mesothelioma Center, a novel immunotherapy drug called ramucirumab significantly…
Aridis Pharmaceuticals has released the data from the first phase of its clinical trial of AR-501. This treatment is inhaled to clear lung infections that are commonly experienced by those…
As you know, COVID-19 has fundamentally changed our daily life. What began as a novel coronavirus in Wuhan, China has developed into a global pandemic: 9 million diagnosed cases…
According to a press release from HemoShear Therapeutics, the company has recently received official clearance from the US Food and Drug Administration (FDA) in regards to its Investigational New Drug…
The Rare Diseases Clinical Research Network (RDCRN) has recently launched a survey aiming to assess the impact of COVID-19 on the rare disease patient community. This initiative is important as…
Cures Within Reach is an organization that aims to help patients by repurposing research. They work towards getting treatments quickly and safely in an effort to aid those who need…
Earlier this year, The Leukemia & Lymphoma Society hosted a presentation by Dr. Eunice Wang, Clinical Leukemia Service, Professor of Oncology, Roswell Park Comprehensive Cancer Center. Dr. Wang’s presentation…
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