The FDA has just announced that they have lifted the temporary hold placed on pheNIX gene therapy. This gene therapy was created by Homology Medicine as an investigational treatment for…
According to a recent article, patients with phenylketonuria (PKU) are unable to eat proteins because of their condition. However, a new probiotic might be the solution. Phenylketonuria (PKU) PKU is…
Unfortunately, clinical trials do not always proceed the way that drug developers - and patients - hope. For example, an ongoing Phase 1/2 clinical trial evaluating BMN 307, an investigational…
Many rare disease patients are familiar with high prices for treatment. In fact, the most expensive medication in the world treats a rare disease: Zolgensa, a gene therapy for spinal…
Gene therapies are becoming an increasingly popular treatment for genetic diseases, especially rare ones. Now, medical professionals are trying a new strategy with gene therapy: adding acetaminophen, also known by…
Nash Hensley is six years old, but in his short life he's already faced many challenges. He was diagnosed at birth with not one, but two rare diseases. First, Nash…
According to a story from express.co.uk, a treatment for phenylketonuria (PKU) is currently under consideration for coverage on the UK's National Health Service (NHS). The treatment is called Kuvan and…
Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) The RDCA-DAP, funded by the FDA, was first launched in September of 2019. Its aim was to make data more accessible for…
On October 2, 2020, biotechnology company BioMarin Pharmaceutical ("BioMarin") announced that its investigational gene therapy candidate BMN 307 received the Fast Track designation from the FDA. The therapy is designed…
BioMarin has been working to develop treatments for phenylketonuria for 15 years, and it is a cause they are very committed to. That is why they are so excited to…
Safety of Sweeteners Sweeteners sound like the ideal solution to added sugar. No calories and none of the cons of refined sugar means you can enjoy it in excess right?…
During the 10th European Conference on Rare Diseases & Orphan Products, hosted by Eurodis, many people advocated for improved newborn screening panels. In particular, these panels should include more testing…
APR Applied Pharma Research is a small pharmaceutical company in Switzerland concerned with innovations for rare diseases. They have recently been granted the Orphan Drug designation by the FDA, meaning…
Rubius Therapeutics is a biopharmaceutical company that focuses on cellular medicines. They have recently dosed the first patient in Phase 1b of their clinical trial of RTX-134. This therapy is…
According to a story from globenewswire.com, the genetic medicines company Homology Medicines, Inc., has recently released promising early data from its phase 1/2 clinical trial. This clinical trial is testing…
According to a story from BioSpace, the genetic medicines company Homology Medicines, Inc., has recently released preclinical data supporting the development of two experimental gene therapies. One, called HMI-202, is…
This is the story of a brave journalist who was determined to improve care for rare disease patients in her country, North Macedonia. North Macedonia is a developing country located…
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