According to a story from Ankylosing Spondylitis News, findings from a phase 3 clinical trial suggest that the drug secukinumab (marketed as Cosentyx) could be an effective treatment for non-radiographic…
You may forgive the Sci-Fi esque headline, but sometimes the truth is stranger than fiction! The 28-year-old from France (identified in this CNN piece as Thibault) was almost completely paralyzed…
According to a story from ir.stockpr.com, the biotechnology company Cyclo Therapeutics, Inc. (previously known as CTD Holdings) has recently thrown its support behind a Niemann-Pick Disease Type C newborn screening…
Hereditary Angiodema Hereditary angioedema (HAE) is a rare disease typically caused by a SERPING1 gene mutation. This protein is responsible for coding for the protein C1-INH. With improper levels of…
According to a story from BioPortfolio, the biopharmaceutical company Collaborative Medicinal Development, LLC has recently announced that the first patient has been enrolled in a clinical trial that will test…
About HAE Hereditary angioedema (HAE) is a rare disease that is caused by the deficiency or reduction of a protein called C1-INH in the body. When this protein is not present,…
According to a story from BioPortfolio, the drug company Octapharma USA sponsored the 71st Bleeding Disorder Conference, which is put on by the National Hemophilia Foundation. The company is also…
BrainStorm Cell Therapeutics has just announced that they have completed enrollment in their Phase 3 clinical trial for amyotrophic lateral sclerosis (ALS). Enrollment was initiated way back in October of…
According to a story from Venture Beat, the company Healx has succesfully raised $56 million in funds that will go towards using artificial intelligence (AI) to identify new potential treatments…
According to a publication from Scleroderma News, the American Food and Drug Administration (FDA) recently granted Breakthrough Therapy status to Ofev (generic name nintedanib), as a treatment for progressive fibrosing…
According to a story from MedCity News, interim data from a phase 1/2 clinical trial testing Zolgensma, a gene therapy for spinal muscular atrophy, in patients between the ages of…
According to a story from PR Newswire, the nonprofit groups Duchenne UK and Parent Project Muscular Dystrophy (PPMD) have jointly awarded grant funding to the tune of $200,000 to Dr.…
Diagnosis, Treatment, and Adherence There are many factors necessary for an individual to be able to thrive with a serious medical diagnosis. First, they must receive the correct diagnosis so…
According to a story from news-medical.net, the National Institutes of Health (NIH) has doled a total of $31 million on grants this year to 20 different research teams. The goal?…
According to a story from med.miami.edu, an international research consortium known as Clinical Research in ALS and Related Disorders for Therapeutic Development (CReATe) has just been awarded a renewal grant from…
EPP Erythropoietic protoporphyria (EPP) is a rare disease that causes extreme light sensitivity. This sensitivity is so extreme that being outside is extremely difficult for patients and many only come…
According to a story from abc.net.au, Darren Bullock is getting pretty long in the tooth---at least for a cystic fibrosis patient. He is 44 years old, and his lungs currently…
Supplies of a Critical Bladder Cancer Drug are Dwindling Bob Field, a 72-year-old New York banking executive, received a call from his urologist’s office canceling his next appointment. According to…
According to a story from ncbiotech.org, a new partnership between Vertex Pharmaceuticals and RNA-based therapeutics company Ribometrix aims to develop potential treatments for serious diseases, including Huntington's disease, which is…
According to a story from PR Newswire, the biopharmaceutical company Clene Nanomedicine, Inc., has issued an announcement in regards to its Australian subsidiary. This subsidiary has been awarded a grant…
According to a recent press release from the Massachusetts-based biotechnology company Avrobio, Inc., the first patient has been dosed in the Company's phase I/II clinical study of their investigational cystinosis…
In a recent press release, Massachusetts-based biotech company Amylyx Pharmaceuticals announced the completion of dosing in the 24 week study of their experimental amyotrophic lateral sclerosis treatment AMX0035. About Amyotrophic…
A Phase 3 trial for Cushing syndrome led by Maria Fleseriu at Oregon Health and Science University has just been completed. It examined a new investigational treatment called levoketoconazole. About the…
Gene therapies have huge potential in the field of rare disease. The fact that these therapies could offer a potential cure otherwise fatal diseases has given families incredible hope. Unfortunately,…
Geisinger Health System is based out of Pennsylvania. They have recently initiated a novel program that has goals to make DNA sequencing a standard part of routine clinical care. Next…
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