According to a story from Trinity News, a team of researchers with the Academic Unit of Neurology at Trinity College Dublin have recently conducted a study that maps the ways…
According to a story from Financial Buzz, the biopharmaceutical company Anavex Life Sciences Corp. has recently announced that the Independent Data Safety Monitoring Board (DSMB) has completed its review of…
Usually when people think of a seizure disorder, they think of something called tonic-clonic seizures or episodes where a person loses consciousness, has muscle stiffness, spasms, and jerky movements.…
According to a story from news.cision.com, the Swedish biotechnology company Cyxone recently announced positive results from the company's phase 1 clinical trial. This trial was testing the tolerability and safety…
According to a story from globenewswire.com, Akcea Therapeutics and Ionis Pharmaceuticals recently announced the release of top line results from a clinical study. This trial tested the drug WAYLIVRA as…
According to a story from globenewswire.com, the drug company Zynerba Pharmaceuticals, Inc. has announced that the data from its phase 2 open label trial has been officially published. This clinical…
According to a publication from Duke University, Dr. Karissa Gable, a neurologist at the University, has received a one-year research grant from the GBS|CIDP Foundation International to study the activity…
According to a story from Connecticut Children's, the hospital is one of the very first in the country to administer Zolgensma, a recently approved gene therapy for the rare genetic…
Lucy is now ten years old and stable, living her life as close to a normal ten-year old as possible. However, she was born with a super rare disease…
According to a story from fda.gov, the US Food and Drug Administration (FDA) has approved the drug pexidartinib (marketed as Turalio) as a treatment for tenosynovial giant cell tumor (TGCT)…
According to a publication from Scleroderma News, a recent study suggests that blood concentrations of certain immune cells could correlate with the severity of cases of systemic sclerosis. About Systemic…
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It's common knowledge among researchers who study diseases called toxic proteinopathies, that this grou of disorders are the result of misfolded proteins that reside in cells. Certain proteins fail to…
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…
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