By: Natalie Homan from In The Cloud Copy Connie Elson was a happy, independent little girl before she started showing worrying symptoms at the age of four. After many doctor…
by Jodee Redmond from In The Cloud Copy Rich Horgan is the founder of the Boston-based biotech non-profit, Cure Rare Diseases. The 28-year-old decided to take action over concerns about…
In an interview with Healio, Dr. Jennifer Miller says that there are signs of hope for improved outcomes in patients living with Prader-Willi syndrome, a rare genetic disorder. Dr. Miller…
If you're like me, news about COVID-19 has been dominating your life recently. The novel coronavirus rapidly spread across the world over the past few months. At the time…
Fabry disease brings symptoms that can affect multiple organs throughout the body. The kidneys and gastrointestinal system are commonly affected, which can make life difficult. According to an article from…
Ibudilast is a drug that has been used to treat asthma, but it is now being evaluated as a treatment for COVID-19. Acute respiratory distress syndrome (ARDS) is a severe…
According to a recent FDA press release, the agency has given its first approval for a drug developed specifically for adult patients diagnosed with upper tract, low-grade, urothelial cancer.…
Ashlyn Sanders was a graduate student at Duke University when she experienced her first seizure. The UNC alumna was shocked. She pursued an MRI, which ended up discovering a…
George and Josie Taylor fell in love in their 20s. 52 years later and happily married, the couple believed that they could conquer anything together. But COVID-19 threw a…
According to Ankylosing Spondylitis News, a recent study has found that TNF inhibitors are often not effective treatments for patients with ankylosing spondylitis. Read the full study in BMC Rheumatology.…
In a post on the Metachromatic Leukodystrophy (MLD) Foundation's website, the organization discusses the possibilities of using gene therapy as a treatment for this rare genetic disorder. The page discusses…
According to a story from Hematology Advisor, the drug luspatercept (marketed as Reblozyl) recently demonstrated its ability to reduce transfusion burden in patients with beta thalassemia that depend on regular…
In a story from Vietnam News, doctors in Hanoi, Vietnam at the Central Paediatrics Hospital announced for the first time that they had performed a successful bone marrow transplant using…
According to a story from Biospace, the RNAi therapeutics company Alnylam Pharmaceuticals Inc. recently announced that its experimental product candidate vutrisiran has earned Fast Track designation from the US Food…
According to an article in Healio, the immunosuppressing drug voclosporin was found to be effective in stimulating renal responses in patients with lupus nephritis. About Lupus Nephritis Lupus nephritis…
According to an article in Science Daily, Professor David Nicewicz and a team of researchers at the UNC Department of Chemistry, discovered a method of creating tracers used in…
As reported in Biospace, a new gene therapy has made notable progress towards curing x-linked retinitis pigmentosa, a disease responsible for sight loss in men. Until now, the disease irreversibly…
The Health Medicine Network has shared that researchers found a potential early biomarker that could help track the development of non-alcoholic fatty liver disease. With up to 80% of…
A press release from the US Food and Drug Administration (FDA) recently announced the approval of the drug selumetinib (marketed as Koselugo) for the treatment of patients aged two years…
According to a story from XTalks, pulling off a clinical trial testing a new drug in a scientifically rigorous manner is no simple feat. It requires the triangulation of a…
According to a story from globenewswire.com, the biopharmaceutical company Liquidia Technologies, Inc. just announced that the company's New Drug Application (NDA) has been officially accepted by the US Food and…
According to a story from Malvern Daily Record, the biopharmaceutical company Millendo Therapeutics, Inc. recently released data from its phase 2b clinical trial. This study was testing the company's experimental…
As originally reported in Biospace, Cushing disease, a disease due to excessive cortisol, has recently had its first ever treatment option approved by the FDA for adult patients who are…
According to a release on Biospace, the European Commission (EC) has approved Takeda Pharmaceutical's ALUNBRIG (brigatinib) as treatment for patients with anaplastic lymphoma kinase-positive (ALK+) advanced non-small cell lung cancer…
The drug levodopa and most other therapies developed over the years for Parkinson's disease have been centered on correcting the lack of dopamine. Researchers acknowledge that the reason dopamine…
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