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…
A recent press release from Fulcrum Therapeutics, a Massachusetts-based biotechnology company, announced preliminary findings of the company's phase 1 clinical study of losmapimod in the treatment of facioscapulohumeral dystrophy. Losmapimod is…
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…
Check out Part One here! Sean Baumstark and Kyle Bryant, hosts of the 2 Disabled Dudes Podcast, led a panel discussion with Neena Nizar, Onno Faber, and Katie Stevens. Kyle…
Sometimes, rare diseases are so rare that they are just as unique as the individual who has it. One child was suffering from a fatal neurodegenerative disease, and the family…
This week, many cities and regions across the country acknowledged Indigenous Peoples' Day, a day to remember and honor the histories and cultures of Native Americans. The treatment of Native…
According to a story from BioSpace, a global collaborative called the Trajectory Analysis Project recently released a study that utilized the North Star Ambulatory Assessment (NSAA), a recently developed measurement…
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…
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According to a recent article published by the University of California San Francisco, Pelizaeus-Merzbacher disease (PMD) is a rare neurological disease affecting young boys. PMD fatalities may occur before the child…
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…
The 2019 Global Genes Rare Patient Advocacy Summit was filled with incredible presentations that highlighted steps for progress in research and advocacy organization, provided solidarity for the rare community, and…
As originally reported by EurekAlert, eosinophilic gastrointestinal disorder research will receive a boost with the renewal of a $7.57 million grant administered over five years awarded to the Cincinnati Children’s…
A new joint clinicial investigation has come out with a reminder to patients with primary hyperoxaluria type 2 (PH2) to adherr to the requirements regarding careful follow up proceedings. This…
The drugs for current inflammatory bowel disease (IBD) patients only help around 50% of patients. And some who do find relief find it short lived as remissions often occur.…
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…
According to a story from curetoday.com, Betty Strommer had been a breast cancer survivor for years when, in 2013, she was suddenly diagnosed with cancer again. This time, it was…
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…
According to a story from the Cystic Fibrosis Foundation's Community Blog, the internet allows for access to a trove of knowledge and information literally at your fingertips. This has naturally…
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,…
According to a story from offspring.lifehacker.com, medical abnormalities and conditions that affect the skin are often something that many of us will notice on our own. Once a skin problem…
According to a story from Scope, Dr. Megan Mahoney has been spearheading a project with Stanford Medicine that aims to transform the dynamic between patients and their care providers. This…
One of our partner organizations, the MDS Foundation, is hosting another free MDS Patient & Family Forum in Dallas, Texas. When: Saturday, November 9th, 2019 Where: Aloft Hotel Dallas Love Field 2333…
A New Collaboration The National Hemophilia Foundation (NHF) is a nonprofit specifically focused on accelerating research for Hemophilia, a rare blood disorder. It was established in 1948 and now has…
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