According to a story from IRB Barcelona, a recent conference has brought together researchers from around the world that are dedicated to the study of the cell cycle and two…
According to a story from Business Wire, the company Alexion Pharmaceuticals recently announced that it has submitted a Biologics License Application (BLA) to the FDA for the approval of one…
According to a story from CheckOrphan, Express Scripts has prepared a Rare Conditions Care Value (RCCV) program. This program is designed to help make the diagnosis and treatment process for…
Happy start-of-summer everyone! We're kicking off the summer with a wrap up on rare disease news this week. We have an article about a systemic mastocytosis study, and a clinical…
According to a story from The Spectator, Madeline Pitts, who has two forms of terminal cancer, will have June 9th be dubbed "Maddie Day" in her honor. She is thirteen…
According to a story from apnews.com, Mike Coble, a senior at Byron High School in southeastern Minnesota, was recently diagnosed with Guillain-Barre syndrome. Mike is on three different sports teams…
According to a story from tdn.com, the radio host Jamie Dupree will soon return to work after nearly a two year absence. However, he will not be doing any announcing…
According to Financial Buzz Chiasma, Inc. has reached a new clinical stage in its development of a treatment for acromegaly. The trial, known as CHIASMA OPTIMAL, tests a treatment currently…
On June 28th, 2018, the National Society for Phenylketonuria (NSPKU) is holding a special event that is meant to challenge Members of Parliament (MPs) across the UK. This challenge is…
Data from a Phase II clinical trial of the experimental treatment reldesemtiv for the treatment of spinal muscular atrophy has been announced. The drug has shown positive effects in the…
According to a story from globenewswire.com, the corporation Retrotope recently announced the initiation of single-patient, expanded access trials of its experimental product RT001. The patients involved have familial encephalopathy with neuroserpin…
According to Pulmonary Fibrosis News, a study with mice showed that a certain compound found in plants, parthenolide, is associated with a sizable reduction in lung fibrosis and the subsequent inflammatory…
An experimental gene therapy designed to treat Duchenne muscular dystrophy has produced exciting preliminary results in a Phase 1/2a clinical trial. Sarepta Therapeutics, the company behind the therapy’s development, announced…
An open-label extension of a Phase II study has shown that the experimental drug lenabasum is fairly safe and tolerable in patients who have diffuse cutaneous systemic sclerosis. The full…
According to a story from Newswise, a team of scientists and researchers have recently completed research that managed to reveal the genetic nature of the unusual rare disease neuromyelitis optica.…
According to a story from UK Healthcare, a team of researchers and scientists at UK Healthcare is preparing to start a clinical trial that will test a new therapy aimed…
Lisa Ray starred in the 2005 film Water. She also appeared in TV shows such as Endgame, Psych, and Top Chef Canada. Now, as an advocate for people with multiple…
The experimental gene therapy LentiGlobin for the treatment of severe sickle cell disease is being investigated in an ongoing Phase 1 clinical trial. Bluebird Bio, the company developing the therapy,…
Clinical data from a Phase 2 study of the experimental drug X4P-001-RD for the treatment of WHIM syndrome has been announced at the European Hematology Association’s 23rdCongress in Stockholm. The…
According to a story from BioPortfolio, the corporation Herantis Pharma Plc recently announced that its Phase 2 clinical trial measuring the effectiveness of Lymfactin, a gene therapy, in the treating…
According to a story from the Ottawa Sun, several hundred people participated in the annual ALS Walk on June 9th, in which people walk down the Sir John A. MacDonald…
According to a story from globenewswire.com, the biopharmaceutical company Idera Pharmaceuticals recently released the data from its Phase 2 trial for IMO-8400, an investigational product in development for the treatment…
According to a story from PR Newswire, the biotechnology company REGENXBIO recently announced that the U.S. Food and Drug Administration (FDA) has granted its experimental gene therapy product RGX-111 Fast…
According to Bright Surf, a recent study has led researchers to believe that children with neuroblastoma are at a higher risk of developing long-term psychological impairment. Neuroblastoma is a rare…
The first patient has undergone stem cell transplant treatment in the third cohort of a Phase I clinical study of the experimental treatment ISC-hpNSC for Parkinson’s disease. The full article can…
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.
