Gaucher disease (GD) is a rare lysosomal storage disorder. It is caused by a deficiency in the glucocerebrosidase enzyme. In June, a new study was published in the International Journal of…
According to a press release from BeiGene, the Company and SpringWorks Therapeutics have launched MapKure, LLC — a new, jointly-operated venture owned by both partners. MapKure was founded to develop…
According to a story from PR Newswire, the drug selinexor (marketed as Xpovio), which was developed by Karyopharm Therapeutics, was recently approved by the US Food and Drug Administration (FDA)…
Growing up "Normal" Growing up, Steph Derham always thought she was normal. She says she has her parents to thank for that. Steph is incredibly grateful that her mom and…
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Duchenne muscular dystrophy (DMD) is the most common, yet severe, form of muscular dystrophy, a group of rare neuromuscular disorders. DMD is caused by mutations in the gene that codes…
According to a publication from Fabry Disease News, Russian researchers recently screened over five-and-a-half thousand patients for Fabry disease while they continued with prescribed hemodialysis. The researchers' study, published in…
Rare Disease Fund Many families affected by rare diseases cannot afford the necessary treatments. Due to the small population of people these diseases affect, exorbitant price tags are often placed…
As reported in RadioFreeEurope, Russian biologist Denis Rebrikov is currently challenging the norm of caution regarding gene-editing in the science world, with efforts to gain the go-ahead from Russia to…
Camp Notaclotamongus In Eureka, Missouri, Camp Notaclotamongus has some typical activities for a summer camp. They state that camper goals aim to build friendships, confidence, and personal growth with outdoor…
ABC News and Good Morning America brightened up our morning by featuring a story of 1-year-old Alle Jilg, whose life was saved by total stranger's bone marrow donation. Alle is…
July 13th is GIST Awareness Day! Let's learn a little more about this rare form of cancer, and then see what we can do is raise more awareness! What are…
This bit of 2017 news flew under the radar! But it's worth shining a spotlight! In 2017, Macedonia became the first country in the WORLD to issue a postage stamp…
According to a story from Market Screener, the biopharmaceutical company Theranexus and the Beyond Batten Disease Foundation (BBDF) have recently signed an agreement to work towards the continued development of…
According to a story from prnewswire.com, the biopharmaceutical company Aridis Pharmaceuticals, Inc. has recently announced that the US Food and Drug Administration (FDA) has awarded Orphan Drug designation to the…
The FDA has approved a new therapy for chronic immune thrombocytopenia (ITP). This treatment is an oral therapy to be taken with food, created by Dova Pharmaceuticals. It is called…
Frankie Jr. Muretic Frankie Jr. Muretic lives in Mississauga in Ontario, Canada. One morning he woke up with a rash, causing his parents concern. They brought the five-year-old boy to…
According to a story from news-medical.net, a team of scientists affiliated with the Swedish Karolinska Institutet have developed a new method for categorizing patients with the rare inflammatory bowel disease…
According to a story from Japan Today, a recent ruling from a Japanese court has ordered the government of that country to pay damages to the tune of 370 million…
July is hemochromatosis awareness month - and in its honor, below is some useful information that could provide helpful. One thing to remember about Hemochromatosis is that early detection is…
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The FDA is putting patients with rare diseases front and center knowing that these patients have little support and in many cases cannot find specialists to diagnose and treat their…
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Dr. Tracy Grikscheit is a leading surgeon in the field of tissue engineering. Dr. Grikscheit, together with her colleagues at Children’s Hospital Los Angeles, co-authored an article for the…
Seems like we JUST had a presidential election, right? But we're in the dead of summer 2019, inching closer to a presidential election that will once again take over our…
Happy Fourth of July Season! Today, we're highlighting stories about research and social media, robots in caregiving, sisters carrying each others' babies, and AI advancing glioblastoma research. After this past…
Dicerna Pharmaceuticals has just announced that they have submitted a Clinical Trial Authorization application for their investigational therapy DCR-A1AT. This application has been submitted to the Swedish Medical Products Agency…
According to a publication from the Basingstoke Gazette, a Basingstoke family is raising money for the children's hospice center that supported them through the loss of a child. Jessica and…
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