An online patient survey has found that 96% of pulmonary fibrosis patients, and 93% of cystic fibrosis patients, support being able to monitor their lung function at home between visits…
According to a recent study, cognitive behavioral therapy may be the solution for patients with misophonia, The Daily Beast reports. Misophonia, which literally means “hatred of sound,” is a rare…
Eva-Rose Sturgess is eighteen months old. She faces a disease for which there is no known cure: leukodystrophy. Her parents felt like their world came apart at the seams when…
According to a story from PR Newswire, the pharmaceutical company Therapix Biosciences recently announced the results of a Phase IIa study for its drug candidate THX-110. The therapy is indicated…
According to a story from adc.net.au, an innovative new cancer treatment regimen is responsible for doubling survival rates for patients with certain types of rare, difficult-to-treat blood cancers. This combination…
The U.S. Food and Drug Administration (FDA) have just approved a drug called Afinitor DISPERZ for treating tuberous sclerosis complex-associated partial onset seizures, reports PR Newswire. This makes it the…
According to a story from QQ Malaysia Online, patient advocates are looking for ways to raise awareness of rare diseases in Malaysia and get help to forgotten patients that desperately…
Patients with sickle cell disease often face stigma about their need for pain medication because care providers are suspicious of drug abuse, reports The Dallas Post Tribune. Although this is…
According to a story from The Telegraph, a team of Chinese and American scientists have used gene editing to create pigs with Huntington's disease. A portion of human genetic material…
According to a story from PR Newswire, the science and tech company Merck recently announced that Mavenclad (cladribine) was recently approved for public use by the UAE Ministry of Health…
Now these are the kind of stories we love to read about! Canadian Revée Agyepong is the first adult in Canada to be cured of sickle cell anemia through stem cell…
According to a story from ankylosingspondylitisnews.com, a significant proportion of patients stopped taking a biosimilar for infliximab within six months of transitioning to the new medication. This suggests that the…
According to a story from genengnews.com, the pharmaceutical company Solid Biosciences is pledging to continue developing its investigation drug candidate SGT-001 for Duchenne muscular dystrophy. This is despite its clinical…
Tyler Perry’s Madea may have saved Boone Begner’s life. Born with heterotaxy syndrome, Boone’s heart works with only three chambers instead of four. The condition often becomes extremely dangerous to…
According to a story from wave3.com, time was running out for Leyan Albazzour, who is nine years old. Born in the Palestinian city of Ramallah, the treatment for her Apert…
According to a story from Neurology Advisor, a retired physician experienced the challenges of being a rare disease patient first hand after being diagnosed with primary periodic paralysis thanks to…
According to a recent story from pm360online.com, the pharmaceutical company Shire announced that the marketing application for its drug product lanadelumab has gained approval from the European Medicines Agency (EMA).…
According to a recent story from CityNews Toronto, Alara Ozkose's Dravet syndrome was preventing her from being able to live a life that was even close to normal. She was…
According to a story from Newswise, researchers at the University of Texas Southwestern Medical Center are reporting the a new drug developed for the treatment of rare, pediatric cancers, has…
According to a story from mrknewsroom.com, the pharmaceutical companies Merck and AstraZeneca recently announced that the marketing application for the investigational drug olaparib (brand name Lynparza) was recently certified for…
According to a story from the Independent, father Nick Sireau is worried that Brexit, the United Kingdom's plan to withdraw from the European Union, could affect the ability of his…
According to an announcement from the pharmaceutical company Retrophin, Inc., the company is starting to move forward with phase 3 trials for its drug candidate sparsentan. The drug is in…
According to a story from biopharmadive.com, the pharmaceutical company Roche is reporting a total of five deaths of patients with hemophilia A that have taken the company's product Hemlibra. While…
According to a story from 4-traders.com, the pharmaceutical company SteroTherapeutics recently announced that its drug candidate ST-002 was awarded Orphan Drug Designation by the U.S. Food and Drug Administration (FDA).…
The United States Food and drug Administration recently announced its decision to grant a priority review. The privilege of expedited process, according to a press release, falls to AstraZeneca and…
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