According to a story from MedCity News, a patient with the rare blood disorder beta thalassemia will be the first patient dosed in a Phase I/II clinical trial involving a…
Spinal Muscular Atrophy Spinal muscular atrophy (SMA) is caused by a mutation in the SMN1 gene. This mutation results in a loss of motor neurons causing weak and atrophied muscles.…
A diagnosis means everything for a rare disease patient. However, to process 200 patient records manually, it would take the most dedicated physician approximately 40 hours. That's one full work…
According to a story from EurekAlert!, a collaborative team of researchers from the University of Florida and The Salk Institute are using cryo-electron microscopy in order to more closely examine…
The speciality pharmaceutical company Advicenne has announced that the US FDA has cleared the Investigational New Drug (IND) application submitted on behalf of ADV7103. ADV7103 is an investigational treatment for…
Many people are familiar with Carl Zimmer as a science columnist from The New York Times. His columns and research dug deeply into the field of genetic research and gene…
The US Food and Drug Administration (FDA) has just awarded Breakthrough Therapy Designation to a drug (OMS721) being developed to treat thrombotic microangiopathy that has developed as a result of haematopoietic stem…
According to a story from Charcot-Marie-Tooth Disease News, the U.S. Food and Drug Administration (FDA), under the leadership of commissioner Scott Gottlieb, has begun to change clinical trials by accepting…
© 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.
