by Lauren Taylor from In The Cloud Copy Huntington’s disease is an inherited disease that slowly causes the breakdown of the nerve cells of the brain, resulting in various disorders…
by Danielle Bradshaw from In The Cloud Copy It has been found that fingolimod - a drug typically used for multiple sclerosis - may also be effective in treating ANCA-associated…
By Lauren Taylor from In The Cloud Copy Myasthenia gravis is an autoimmune condition characterized by muscle weakness and fatigue of muscles that are under the patient’s voluntary control. This…
Salk Institute scientists have scored a significant gain in their quest to find a functional treatment for type 1 diabetes. According to an article in Salk News, scientists at the…
by Lauren Taylor from In The Cloud Copy Duchenne muscular dystrophy (DMD) is a rare genetic condition that ultimately leads to the wasting away of muscles over time. The condition…
Editas Medicine has developed a sickle cell disease (SCD) treatment, and it has recently received the Rare Pediatric Disease designation from the FDA. This means that their therapy, EDIT-301, is…
According to a recent article in FierceBio, Andrea Mazzocchi, Known Medicine CEO, is not trying to reinvent the wheel. On the contrary, her company will be providing a tool to…
Last week, biopharmaceutical company Deciphera Pharmaceuticals announced results from its Phase 1 clinical trial on QINLOCK (ripretinib). The study, published in the Journal of Clinical Oncology, tested QINLOCK as a potential…
According to MedCity News, a third patient dosed with AT132 during the ASPIRO trial has died. The Phase 1/2 clinical trial tested the efficacy, safety, and tolerability of AT132 for…
BioMarin Pharmaceutical Inc. has recently submitted a new drug application (NDA) for their achondroplasia treatment, vosoritide. This once a day injection is for children who live with the disorder, and…
The World Orphan Drug Conference USA 2020 The Global Orphan Drug Conference and Expo August 24th - August 26th, 2o20 in National Harbor, MD The conference is an event that…
A study conducted at the University of Texas has discovered that combination therapy may be a viable treatment for cholangiocarcinoma with BRAF mutations. Researchers treated participants with both dabrafenib and…
Sometimes, researchers find beneficial medications during clinical trials. Other times, the therapy just falls short. According to Myasthenia Gravis News, the latter is true for Firdapse (amifampridine phosphate). The…
This past week, biopharmaceutical company Mustang Bio announced that their gene therapy candidate MB-107 received Rare Pediatric Disease Designation. The gene therapy candidate is designed to treat X-linked severe…
To capture the essence of palliative care, the World Health Organization defines it as the prevention and relief of suffering associated with a life-threatening illness. An article in Parkinson’s…
I-Mab, a biopharmaceutical firm, has begun dosing participants in Phase Ib of their trial of plonmarlimab, a rheumatoid arthritis (RA) treatment. Developers of the medication believe that it could become…
A press release issued recently by Regeneron Pharmaceuticals heralded the acceptance of a Biologics License Application by the FDA for Priority Review of its investigational drug, evinacumab. The Priority…
by Danielle Bradshaw from In The Cloud Copy Gastroparesis is a condition in which your stomach’s muscles aren’t able to completely move food through your stomach. Symptoms of gastroparesis include…
by Lauren Taylor from In The Cloud Copy Pediatric pulmonary arterial hypertension is a disease in which the blood flow leaving the right side of the heart faces increased resistance…
According to BioSpace, the first patient has been enrolled in a trial of pamrevlumab, a Duchenne muscular dystrophy treatment. The third phase of LELANTOS plans to evaluate the drug's effects…
Arrowhead Pharmaceuticals has began dosing patients in its AROENaC1001 trial, which evaluates ARO-ENaC as a treatment for cystic fibrosis. 54 patients will be treated with this therapy in an effort…
This week, Regeneron Pharmaceuticals announced that their Biologics License Application (BLA) for evanicumab, a treatment for homozygous familial hypercholesterolemia (HoFH), was granted Priority Review status by the FDA. The…
According to a story from curetoday.com, patients living with mantle cell lymphoma, a rare form of blood cancer that makes up only around six percent of diagnosed non-Hodgkin lymphoma cases…
Corbus Pharmaceuticals has just announced that participant enrollment for their Phase 3 trial of lenabasum for dermatomyositis has been completed. Dermatomyositis Dermatomyositis (DM) is a rare autoimmune disease. It can…
Would you be shocked to find out that before Evrysdi, there were zero orally-administered drugs approved to treat patients with spinal muscular atrophy (SMA)? But now, things have changed. Last week,…
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