Anna is from England and recently finished her undergraduate degree. She has an interest in medicine and enjoys writing. In her spare time she likes to cook, hike, and hang out with cats.
An international team of researchers studying the effects of progressive familial intrahepatic cholestasis (PFIC) in children have found that half of the children had received a liver transplant by age…
Continue ReadingHalf of Children with PFIC will Receive a Liver Transplant by Age 10, Study Finds
The Vasculitis Foundation has released a video called “Connect the Dots” to raise awareness about vasculitis within the medical community, reports Newswise. Vasculitis is a frequently misdiagnosed or undiagnosed condition,…
Continue ReadingIt’s Vasculitis Awareness Month 2018, and a Video has Been Released to Educate Doctors About the Condition
A rare but serious disease caused by flesh-eating bacteria has been studied, and researchers have identified the mechanism by which it progresses. This has lead them towards potentially effective treatments…
Continue ReadingResearchers Have Discovered a Mechanism and Potential Treatments for Necrotising Fasciitis
The family of Tessa Jowell, a UK politician who recently passed away from a brain tumour, have commended the government for their commitment to improving diagnostic and treatment services for…
Continue ReadingThe UK Government Commits to Improving Brain Cancer Services in Memory of Tessa Jowell
A phase 3 Murano clinical trial has shown that combining the drugs Venclexta (venetoclax) and Rituxan (rituximab) could be an effective treatment for chronic lymphocytic leukaemia, reports Cancer Updates, Research,…
Continue ReadingA New Drug Combination May Provide an Effective Treatment for Chronic Lymphocytic Leukemia
Patient Worthy recently attended ALD Life’s Community Weekend 2018, which brought together people and families with Adrenoleukodystrophy (ALD) and Adrenomyeloneuropathy (AMN), and scientists, researchers, and doctors. One of the speakers…
Continue ReadingUnderstanding the ‘Building Blocks’ of ALD Patient Care
The U.S. Food and Drug Administration (FDA) has just approved a drug called Darzalex (daratumumab) in combination with the drug Velcade (bortezomib), melphalan, and prednisone (known collectively as VMP) for…
Continue ReadingA Drug For Multiple Myeloma has Been Approved by the FDA
The hashtag #hotpersoninawheelchair went viral after the Youtuber and activist Annie Segarra used it as a response to an offensive tweet by Ken Jennings, reports The Guardian. Jennings, best known…
Continue ReadingA New Twitter Hashtag is Challenging Ableist Attitudes
William Eames, a five-year-old boy from a village in Hertfordshire, England, was diagnosed with Duchenne muscular dystrophy when he was three. Since then, his family have worked hard to raise…
Continue ReadingThe Mother of a Boy with Duchenne Muscular Dystrophy Shares Her Family’s Experience with the Disease
A website called CrowdMed is changing the way patients can be diagnosed. It uses a group of people to collectively analyse challenging medical cases through ‘crowd wisdom.’ A TedMed talk…
Continue ReadingCrowdMed Offers Patients a New Route for Diagnosis
A condition called XLHED that was thought to be incurable has been treated, and a paper describing the procedure, which can be found here, was released on the 26th April…
Continue ReadingFetuses with XLHED Have Successfully Been Treated in the Womb, in the First Procedure of its Kind
The first patient has just been dosed in a Phase 3 trial of a drug to treat spinal muscular atrophy, reports Benzinga. The drug, called AVXS-101, is a gene therapy…
Continue ReadingA Phase 3 Trial For a Spinal Muscular Atrophy Gene Therapy Has Just Dosed Its First Patient
The UK government is being petitioned to hold a referendum on the decriminalisation of the drug cannabis. This has implications for people with diseases that may be helped by using…
Continue ReadingThe UK Government is Being Petitioned to Hold a Referendum on Cannabis
Acceleron Pharma has just received Fast Track designation from the US Food and Drug Administration (FDA) for their experimental treatment ACE-083 designed to treat patients with facioscapulohumeral muscular dystrophy (FSHD).…
Continue ReadingAn Experimental Treatment for FSHD Has Been Awarded Fast Track Designation by the FDA
A study has just been published in the New England Journal of Medicine (NEJM) that found that the drug Brineura slows down the deterioration of language and motor function in…
Continue ReadingStudy Finds That an Enzyme Therapy for Batten Disease is Effective
A large clinical study will be carried out to develop a protocol for identifying a rare primary immunodeficiency disease called WHIM syndrome in patients, reports BioPortfolio. The Jeffrey Modell Foundation,…
Continue ReadingA New Study Aims to Improve Diagnoses for People with WHIM Syndrome
Phillippa Farrant, whose son, Daniel, has been diagnosed with Duchenne muscular dystrophy (DMD), recently appeared in Parliament in the UK to fight for improved access to care and treatment for…
Continue ReadingThe UK Parliament Met With a Mother Advocating For Duchenne Muscular Dystrophy Patients
Doctors haven’t been able to work out the cause of a young woman’s medical symptoms, and now the New York Times is appealing to the public for suggestions. This article…
A gene therapy being developed as a treatment for Sanfilippo syndrome has just been awarded Regenerative Medicine Advanced Therapy (RMAT) status in the US, reports GlobalGenes. Sanfilippo syndrome is a…
Continue ReadingA Gene Therapy Being Developed for Sanfilippo Syndrome Has Been Granted RMAT status in the US.
A clinical trial that gave children with progeria an experimental drug that was initially intended for cancer patients has produced promising results, reports The Washington Times. The study found that children…
Continue ReadingAn Experimental Drug May Increase Lifespan in Progeria Patients
The Australian government will subsidize a new treatment for Hodgkin lymphoma, bringing its price down from $200,000 per course to $39.50 for most people, and $6.40 for concession patients, reports…
Continue ReadingA $200,000 Cancer Treatment Will Cost $40 in Australia, Thanks to Government Subsidy
A new study reported on by Hematology Times has found that being taught art can significantly decrease pain and anxiety, and promote a positive mood in patients with hematologic malignancies.…
The US Food and Drug Administration (FDA) has just awarded Breakthrough Therapy Designation to a drug (OMS721) being developed to treat thrombotic microangiopathy that has developed as a result of haematopoietic stem…
Continue ReadingA Treatment for Thrombotic Microangiopathy Has Been Awarded Breakthrough Status by the F.D.A.
A gene therapy called ABO-202 that is being developed to treat Batten disease has just been granted Orphan Drug Designation by the European Medicines Agency (EMA), reports BioPortfolio. This follows…
Continue ReadingA Gene Therapy Being Developed to Treat Batten Disease Has Been Granted Orphan Drug Status in Europe
The US Food and Drug Administration (FDA) has just approved the drug Vonvendi for use in adult patients with von Willebrand disease who are undergoing surgery. This is an extension…
Continue ReadingThe FDA Approves a Treatment for Patients with Von Willebrand Disease Undergoing Surgery
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