According to a story from drugs.com, the biopharmaceutical company Mirum Pharmaceuticals recently announced that they have begun dosing the first patient in a phase 3 clinical trial. This clinical trial…
According to a publication from Ankylosing Spondylitis News, a recent study published in BMC Musculoskeletal Disorders suggests that LINC00311, a lengthy RNA molecule, is over-expressed in individuals with ankylosing spondylitis.…
According to a publication from Hemophilia News Today, an international team of researchers with members from France and the United Kingdom recently conducted a study of over 100 hemophilia patients…
According to a publication from OncLive, the Food and Drug Administration (FDA) recently granted priority review status to a supplemental biologics license application for Zejula. Zejula (generic name niraparib) is…
As reported in Biospace, xpovio (selinexor), a drug used to treat relapsed refractory multiple myeloma, received accelerated approval by the Food and Drug Administration this July. About Multiple Myeloma Multiple…
Kratom, an herbal supplement of the caffeine family used to ward off pain for chronic pain conditions, has received large grants for further research. While many consumers claim the supplement…
According to a story from Scleroderma News, a recent study has identified a host of risk factors that can increase a patient's likelihood of experiencing progressive organ damage. The identification…
According to a story from Ankylosing Spondylitis News, a recent study has found that the class of drugs known as tumor necrosis factor (TNF) inhibitors may actually be safer for…
It was announced this Monday that Fulcrum Therapeutics, a Phase 1 biotech developing therapies based on gene regulation for rare diseases, announced terms for its initial public offering (IPO), aka its…
Our partner, The MDS Foundation is sponsoring another free MDS Patient & Family/Caregiver Forum. The event will take place in Iowa City, Iowa on Saturday July 20th, 2019. Event Summary:…
According to a publication from Business Wire, the Israeli biotechnology company Minovia Therapeutics recently finished dosing the first participant in a phase I/II clinical study of the Company's experimental mitochondrial…
According to a story from Wellcome Center Mitochondrial Research, a multi-center collaborative study that involved researchers from different sites around the UK has resulted in an improved understanding of the…
According to a publication at Markets Insider, the Food and Drug Administration (FDA) recently approved Soliris (eculizumab) as a treatment for neuromyelitis optica spectrum disorder. The approval marks the first…
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Gov.UK recently published a speech by Baroness Blackwood in which she announced that the National Health Service (NHS) is currently involved in one of the most significant transformations in social…
According to a publication from the Oklahoma Nursing Times, Kenneth Wyatt celebrated his 80th birthday this past June 4th. The happy occasion also marked almost five months exactly since Wyatt…
According to a publication from WCAX, care providers at Children's Hospital Colorado are devising new ways to make life with eosinophilic esophagitis easier. One patient, six-year-old Eliana Yelpaala, is already…
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…
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