University of Illinois Researchers have uncovered novel information about pulmonary fibrosis. Their research demonstrates that fibrosis in the lungs can be reversed, and tissue can be repaired using microgel-coated mesenchymal…
States are not able to alter patent protections or set prices but Colorado is no longer waiting for the federal government to act. According to a recent article in…
WLNS Brooklyn Michigan is sharing news about Jaxon Meschke, a four-year-old boy with a rare disease called cystinosis. Jaxon’s body has been fighting the disease since he was six…
Ahead of Cystic Fibrosis Week in the UK, 37-year-old Marc Cotterill, who lives with the disease, has given a video presentation to the European Cystic Fibrosis Conference on the theme…
Researchers from the University of Lausanne and the Wellcome Sanger Institute have just published a new study in Molecular Systems Biology which uncovered how DNA can overcome genetic changes which are…
According to a story from eminetra.com, the month of May was Lyme Disease Awareness Month, which is a suitable time for it as May is often one of the peak…
According to an announcement from the US Food and Drug Administration (FDA), the agency has recently approved a new therapy for Alzheimer's disease. While this is a significant moment for…
A 2019 Survey Found 70% of Patients with NMOSD Relapsed Every Year. According to a recent article in China Daily, the patients interviewed for this article said that their…
The month of June is recognized as CDKL5 Awareness Month, a time set aside to spread awareness about CDKL5 deficiency disorder among the general public and the medical community. June…
The month of June is recognized as Scleroderma Awareness Month, a time for spreading awareness about the rare disease scleroderma in the medical community and among the general population. This…
Rare disease research is often lacking due to a lack of funding, interest, or awareness. To combat this, the Barth Syndrome Foundation (BSF) and American Heart Association (AHA) have joined…
A press release by MYND Sciences announced that the drug research and development company, MYND Life Sciences Inc. has their new intellectual patent for their method of finding biomarkers to…
According to an article in the Times Union, a group of parents with children who have been diagnosed with rare diseases are leading the crusade to foster federal legislation which…
Sobi as well as the Hellenic Institute for the Study of Sepsis have just announced successful results from their SAVE-MORE study for those with COVID-19. The results were from 28…
According to a story from Medscape, US Food and Drug Administration (FDA) has recently issued a new restriction on the use of obeticholic acid (marketed as Ocaliva). The agency now…
BeiGene has just announced that China has given conditional approval to Pamiparib, a PARP inhibitor used as a therapy for BRCA associated ovarian cancer, fallopian tube cancer, and primary peritoneal…
Enzyvant has just announced that they have resubmitted their application of RVT-802 to the FDA as a therapy for pediatric congenital athymia. Their first application was submitted in 2019. The…
Pfizer Rare Disease is an approach taken by pharmaceutical giant Pfizer to develop treatments for rare and orphan diseases. In the past, the company has worked with the National Newspaper…
According to a recent article in MedCity News, families with children who have a rare disease wait on average for five years before receiving an accurate diagnosis. It is…
Rare disease research faces many obstacles: lack of participants, lack of funding, and lack of interest are just a few. Now, the charity Cerebra has launched a network to take…
Mustang Bio, a biopharmaceutical company, and City of Hope, a cancer center, have just revealed that their first participant in their Phase 1 clinical trial for leptomeningeal brain tumors including…
The month of May is recognized as PKU Awareness Month, a time of year set aside to focus on spreading awareness about phenylketonuria (PKU) among the general public and in…
According to a story from republicworld.com, the medical fraternity of Queen Mary's Hospital in King George's Medical University, Lucknow was able to successfully treat a woman, age 35, with immune…
The Rare Disease Legislative Advocates (RDLA) held its monthly webinar on May 20, 2021. The primary focus of the webinar was a discussion and overview of drug pricing proposals that…
In a news release from early May 2021, biopharmaceutical company Bold Therapeutics ("Bold") shared that its treatment BOLD-100 received Orphan Drug designation for the treatment of patients with gastric…
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