Seizures are a common symptom of Angelman syndrome (AS) and the cause of these seizures was unknown for a long time. This lack of knowledge made it impossible to…
Focal segmental glomerulosclerosis (FSGS) is a kidney disease that often results in end-stage renal failure. A kidney transplant is the best way to treat this failure. The issue with this…
Falls are a symptom that affect about 60% of those with Parkinson's disease. This symptom can be debilitating, as many injuries can be the result. The University of Bristol…
Copyscape score: 7% A new clinical trial is meant to find treatments that can slow or stop the progression of motor neurone disease (MND). This disease causes progressive weakness and…
BridgeBio Pharmaceuticals has recently announced that their drug, infigratinib, has received the Fast Track and Orphan Drug designations from the FDA. This drug is meant for the treatment of…
The discovery of a mutated gene is shedding light on the severity of kidney disease in those with Joubert syndrome. The BSND gene is called a modifier gene, and…
Prevail Therapeutics is the creator of PR001, which is a gene therapy used for the treatment of Gaucher disease. They expected to begin dosing in the first half of…
There have been many defining factors throughout the past decade, especially in the healthcare industry. These changes, both good and bad, have defined this industry. From the opioid epidemic…
Sandy Patience is a 57-year-old man from Inverness, Scotland, and he has Huntington's disease. His mother and sister also have this disease, so he has seen others who are affected…
AI Therapeutics is a biopharmaceutical company that has created an artificial intelligence platform that is meant to match drugs to new indications. This platform, called Guardian Angel, has matched…
Hyperphagia is an extreme hunger and obsession with food, and it is a symptom of Prader-Willi syndrome. While many of the other effects of this condition have treatments, this…
A diagnosis is something that is not always easy to attain. Without one, people cannot receive the best treatment for their conditions, miss out on a community of others…
ALS is a progressive disease that causes the loss of muscle function, and it often develops very quickly after a diagnosis. Rami Epstein, the CEO of the biopharmaceutical company called…
Hemophilia A is the most common type of hemophilia, accounting for 8 of every 10 cases. Treatment in the past consisted of the administration of concentrated factor VIII through…
Orphazyme, a biopharmaceutical company, has recently released the data from their study of arimoclomol. This product is meant for the treatment of Niemann-Pick disease type C (NPC). Orphazyme specializes…
Herantis Pharma has been conducting a study of their treatment for Parkinson's disease, which is cerebral dopamine neurotrophic factor (CDNF). Phase 1/2 has been completed, and an extension phase…
Treatment for ulcerative colitis differs based on the individual. Where the inflammation occurs, the severity, and the preference of the patient can all influence the treatment. For those with moderate…
GlaxoSmithKline (GSK) has recently completed and released the results of Phase 3 of its study of benlysta. This drug is made for the treatment of lupus nephritis. Their BLISS-LN…
Savara Pharmaceuticals has recently interviewed Dr. Tisha Wang, who is a pulmonologist and critical care physician at UCLA. Dr. Wang is also an advocate for autoimmune pulmonary alveolar proteinosis…
Regeneron Pharmaceuticals has recently announced the results of Phase 2 of their study of pozelimab, which is an experimental treatment for paroxysmal nocturnal hemoglobinuria. Current therapies for this condition…
No healthcare professional wants to hear that a drug being studied in a clinical trial did not meet the endpoints. It means that after countless hours of hard work,…
A new study has been initiated by a digital health company in the United Kingdom called Congenica Ltd. This study focuses on re-annotating genes that may be associated with…
Galectin Therapeutics has recently started to plan the third phase of its clinical trial of belapectin, which treats nonalcoholic steatohepatitis (NASH). They are making refinements based on a conversation…
It is estimated that about 10% of people in the United States have rare diseases. But what makes a disease rare? How does this distinction affect diagnosis and treatment?…
Alpha-1 antitrypsin deficiency (A1AD) shares similarities with asthma, which leads to it often being misdiagnosed as asthma. This misdiagnosis leads to insufficient treatment. Toronto Western Hospital's Asthma and Airways…
© 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.
