More than 20 years ago researchers at Harvard University, led by professor Oliver Pourquié, discovered how the vertebra in chickens first form. Essentially, a 'tick' initiates the formation of a…
By Rachel Whetstone from In The Cloud Copy The Orphan Drug Act of 1983 was created to encourage the development of drugs to help people with rare diseases. Drug companies…
Epithelioid sarcoma is a rare form (accounts for <1%) of soft tissue sarcoma. Until now, there have been no treatments specifically for this patient population. But the FDA has just…
For much of human history, many genetic disorders were accepted as incurable, their rapid onset bound to take patients' lives before they had the chance to be lived. However, the…
SELLAS has just announced that patient screening has begun for a Phase 3 trial for acute myeloid leukemia (AML). The trial is examining the effects of GPS for patients who…
According to a story from the Irish Medical Times, Ireland's Health Service Executive (HSE) has recently announced the extension of a reimbursement agreement between the agency and the pharmaceutical company…
According to a story from The Edge Markets, Dr. Lee Boon Chye, Malaysia's Deputy Minister of Health, has recently announced an initiative to develop a National Framework for Rare Disease,…
According to a story from streetinsider.com, the Joseph Ahmed Foundation and Tyme Technologies, Inc. recently announced that the first patient has been dosed in a phase 2 clinical trial. This…
According to a story from Wind River Wellness Cancer Retreat & Programs, a young woman named Lauren who had previously attended sessions at the retreat center has been living with…
According to an announcement from the Muscular Dystrophy Association, the organization has recently awarded a total of $1,076,232 in MDA Venture Philanthropy (MVP) funding towards the drug company AavantiBio, which…
By Rachel Whetstone from In The Cloud Copy Justin Stufflebean was 27 years old when he died after being taken to Missouri Western Reception, Diagnostic and Correctional Center (WRDCC). He…
A New Phase 3 Trial A new phase 3 study will begin soon this year at Stanford University. Abeona Therapeutics had been waiting on IRB approval to begin the trial,…
According to a story from Biospace, the drug company Horizon Therapeutics plc has recently announced that the US Food and Drug Administration (FDA) has approved the company's drug teprotumumab-trbw (marketed…
By Rachel Whetstone from In The Cloud Copy Peggy Nipper was struggling with polycystic kidney disease (PKD). Her kidneys were beginning to fail after developing numerous cysts. It can often…
According to a video from khou.com, the recent death of a five year old boy in the Houston area from bacterial meningitis has prompted the release of a guideline video…
This past week Justin Bieber publicized some surprising news amidst some fans commenting that he looked like a “drug addict.” Bieber, 25, recently posted on his Instagram that he…
According to a story from globenewswire.com, the biopharmaceutical company SELLAS Life Sciences Group, Inc. just announced that it has started screening participants for its phase 3 clinical trial. The trial…
Medical science made some great breakthroughs in 2019 and the worldnewspulse.com shares just a few of the most impressive captured moments for science and real life patients. Here are three…
According to a story from the Houston Chronicle, a five year old boy named Myles Cheatham was admitted to the Spring Clinic two days after experiencing symptoms of what was…
The Study: MRx0518-I-003 MRx0518-I-003 is a trial investigating MRx0518 as part of a combination treatment for pancreatic cancer, used with preoperative radiotherapy. The trial will evaluate the therapies safety and…
Sickle Cell Disease Sickle cell disease is a rare blood disorder. It is progressive, debilitating, and sadly can be life-threatening. The genetic defect leads patients' red blood cells to be…
Galactosemia Galactosemia is a rare disease which affects the metabolic system. It causes the body to not be able to process galactose properly. Galactose is a simple sugar present in…
According to a story from BioSpace, the US Food and Drug Administration (FDA) has recently approved avapritinib (marketed as AYVAKIT) as a treatment for gastrointestinal stromal tumors in cases when…
According to a story from euronews.com, the drug maker Novartis has announced that it will give away its breakthrough spinal muscular atrophy gene therapy drug Zolgensma to 100 patients each…
Olmsted syndrome is a rare disease most frequently caused by mutations in TRPV3. For some patients, it is caused by MBTPS2 mutations. It is characterized by palmoplantar keratoderma (PPK) as…
© 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.
