According to a story from First World Pharma, the biopharmaceutical company Mustang Bio, Inc. recently announced that the US Food and Drug Administration (FDA) has approved their investigational new drug…
Are we ready for our close-up? The second annual Selections from DISORDER: The Rare Disease Film Festival at Biotech Week Boston will be held at the Takeda Auditorium in Cambridge,…
According to a story from Pharmaceutical Technology, the company Stoke Therapeutics experimental drug candidate, known as STK-001, has earned Orphan Drug designation from the US Food and Drug Administration (FDA).…
RAREsummit19 is a 1 day summit focusing on patient-centricity in rare disease progress. What if we were able to share information with the power to revolutionise the current rare…
According to a publication from NewZNew, the NeuroGen Brain and Spine Institute in India plans on hosting an outpatient stem cell therapy consultation camp in Chandigarh on the last day…
Happy Thursday! This week, we're highlighting an story about a woman with Bechet disease who advocates for herself and others. Next, we have an article discussing the difficulty of caring…
According to a story from the St. Helens Star, Claire Riley, the mother of Kaycee Bradshaw, said in a recent statement that she would not hesitate to move out of…
At just nineteen, Beth often didn’t feel well and regularly had a sickly pallor. When she went to a doctor, the doctor dismissed her and told her she just needed…
According to a story from Direct Relief, when Yusra was just a year old, she began to have mysterious bouts of fever. Her family had no explanation. By age three,…
According to a publication from ScienceDaily, a new study of propranolol suggests explanations for its ability to shrink certain benign tumors in infants and relieve symptoms of hypotrichosis-lymphedema-telangiectasia syndrome. About…
According to a story from BioPortfolio, a team of scientists associated with the University of Zurich and others have made a remarkable discovery that could reveal new information about the…
According to a story from Drug Development Technology, the drug developer Translate Bio has recently reported interim results from its phase 1/2 clinical trial. This trial is testing the company's…
Brenda has both psoriatic arthritis and psoriasis. She's lived with the conditions since she was a child, learning to care for herself and deal with the challenges that arose. By…
According to a story from the Scientific American, new research is revealing the potential of the human microbiome as a method for treating health problems. Research is also continuing to…
According to a story from True Viral News, anyone who has tried to get something done through governmental means are probably familiar with getting rebuffed and redirected at every turn.…
As reported in EurekAlert, an exciting new treatment method has entered the conversation to treat inflammatory bowel disease (IBD). Researchers have successfuly used mice population to show that precision cutting…
According to a story from BioSpace, the biopharmaceutical company Altavant Sciences has recently announced that the first patient has been dosed in a phase 2a proof of concept trial. This…
Groups like Cure SMA, the Cystic Fibrosis Foundation, the Muscular Dystrophy Association, Parent Project Muscular Dystrophy (PPMD), and the National Organization for Rare Disorders (NORD) have a mission: The United States Senate…
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As reported recently in PhysOrg by NASA, researchers aboard SpaceX CRS-18 cargo flight are focusing on what triggers Parkinson’s disease and multiple sclerosis, both neurodegenerative diseases. Researchers suspect that…
As originally reported in The Rheumotologist, phase three trials for new lupus medicine have failed, disappointing the community that had high hopes for the drug anifrolumab. The community knows the…
According to a press release from American biotechnology company Neurogene, the Company has kicked off a sponsorship of Invitae Corporation's "Detect Lysosomal Storage Diseases" program, which will offer genetic testing…
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Doug Lindsay ran track in high school. According to an article published by CNN Health, Doug had big plans as a senior at Rockhurst University in Kansas City where he…
According to a publication from Medscape, the first ever drug targeted for bladder cancer has shown even more impressive results in post-approval study. Erdafitinib was approved on an accelerated basis…
According to a story from EurekAlert!, a recent study has found that vaccinations are not a risk factor for multiple sclerosis. A risk factor is something that could potentially increase…
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While the last few years have seen major advances in all areas of the pharmaceutical industry, some of the newest and brightest talents are now focusing on cell and…
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