When this 20-year-old male patient was serving in the military, he came down with two severe medical issues: pneumonia and adrenal crisis, categorized by an issue with an adrenal hormone…
The world has now hit over 3.5 million diagnosed cases of COVID-19, with 1.2 million cases in the United States alone. Although the pandemic started in December or January, the…
De acuerdo a una historia de PR Newswire, La Organización Nacional de Trastornos Raros (NORD, por sus siglas en inglés) acaba de lanzar un programa de ayuda crítica COVID-19. En…
Saying that COVID-19 has impacted the world might just be an understatement. In fact, COVID-19 changed the way we interact with each other, businesses, and the world around us. But how…
By Danielle Bradshaw from In the Cloud Copy The Swiss-born pharmaceutical company, Novartis, has been given permission by the Food and Drug Administration (FDA) to begin distributing their oral…
Autism spectrum disorder (ASD) affects one out of fifty-nine eight-year-olds in the U.S. A recent article in Medical News Today outlines a study conducted by neuroscientists from the Korea Brain…
By Payel Bhattacharya, living with von Hippel-Lindau syndrome I, Miss Payel Bhattacharya, was an unusual child. I was never concerned with petty things, the trivia, the dregs in life. My…
Recently, Pfizer's Dr. Ian Winburn sat down with Europabio for an interview in honor of World Hemophilia Day, which just occurred on April 17. In his discussion, Dr. Winburn…
By Jodee Redmond from In the Cloud Copy Glioblastomas are tumors that are particularly difficult to treat. They start forming in the glial cells of the brain and spinal cord…
Phase III of the CheckMate -9ER trial has reached its primary endpoint of progression free survival in those with advanced or metastatic renal cell carcinoma. This study was evaluating CABOMETYX…
by Danielle Bradshaw from In The Cloud Copy Hereditary angioedema (HAE) is a rare condition in which a person has swelling beneath their skin (which can occur in any part…
Peptide receptor radionuclide therapy for advanced neuroendocrine tumors that affect pancreas, GI tract by Mike Bassett, Contributing Writer, MedPage Today April 30, 2020 MedpageToday email article The most significant recent…
According to a press release on Global Newswire, the biopharmaceutical company MediciNova has received a Notice of Allowance for a patent application for MN-166, which could be used to treat…
According to an article published in the Orphanet Journal of Rare Diseases, a workshop held on June 23, 2019 in Orlando, FL focused on a difficult subject: improving detection and diagnosis for…
According to Angioedema News, the European Medicines Agency (EMA) is now reviewing berotralstat as a potential therapy to combat severe inflammation attacks in patients with hereditary angioedema. Hereditary Angioedema Hereditary…
A new resource guide has been developed for X-linked hypophosphatemia (XLH) by Prime, a medical education firm. This project has been supported by Ultragenyx Pharmaceutical Inc. The guide provides resources…
According to a news release from Biospace, recent data from the KALM-2 Phase III trial highlighted how Korsuva, an injectable, is beneficial in treating severe itching associated with severe chronic…
Armené and Michael Kapamajian live in LA. Their son Sasoun was diagnosed with severe combined immunodeficiency (SCID). This means that his immune system is compromised and he is extremely vulnerable…
The World Health Organization and its partners recently launched the Solidarity international clinical trial in search of an effective treatment for COVID-19. The rising death toll for a virus that…
The month of May is Huntington's Disease Awareness Month, in which a special effort is made to improve awareness about this devastating rare disease. This year, the Huntington's Disease Society…
Trevor O'Sullivan Trevor O'Sullivan, who lives in Ireland, has cystic fibrosis (CF). He has undergone two liver transplants. He was the 42nd person in the country to have this surgery.…
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 Hannah Witton, a YouTuber with 600,000 subscribers to her sex and relationships channel, got tired of hearing comments that people with disabilities…
According to a story from Buzzfeed News, the ongoing coronavirus/COVID-19 pandemic has put the brakes on critical rare disease research all over the globe, ranging from early, highly experimental studies…
The FDA recently granted Orphan Drug status to CS1, a drug therapy from Cereno Scientific, according to Pulmonary Hypertension News. CS1 is designed to provide safe and effective treatment for…
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