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.
The company Pharnext SA, which is developing the investigational drug PXT3003 for the treatment of Charcot-Marie-Tooth disease type 1A, has agreed upon a paediatric investigation plan for the drug with…
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The European Medicines Agency Has Approved a Paediatric Investigation Plan for an Experimental Treatment of Charcot-Marie-Tooth Disease Type 1A
The Joshua Frase Foundation has released a new eighteen-minute documentary about the family’s fight for more research into gene therapies for myotubular myopathy, the rare condition that Joshua Frase was…
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An Eighteen-Minute Documentary Shows How the Frase Family Have Fought for Research into Myotubular Myopathy
Catabasis Pharmaceuticals Inc. has announced plans for a Phase 3 clinical trial called Polaris DMD. It will investigate the experimental drug edasalonexent for the treatment of patients with Duchenne muscular…
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An Experimental Drug For Treating Duchenne Muscular Dystrophy Will be Studied in a Phase 3 Trial, Catabasis Says
The US Food and Drug Administration (FDA) has awarded Priority Review Designation to glasdegib, an experimental drug designed as a treatment for previously untreated acute myeloid leukaemia in combination with…
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Priority Review Granted to an Investigational Treatment for Acute Myeloid Leukaemia
Results from research into an experimental therapy called SL-401 for the treatment of patients with blastic plasmacytoid dendritic cell neoplasm have been shared at the Congress of the European Haematology…
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Encouraging Results Have Been Announced From a Trial of an Investigational Treatment For BPDCN
Kalytera Therapeutics, Inc. has announced that a meeting with the Centre for Drug Evaluation and Research of the US FDA is planned for Tuesday, 31st July this year. The purpose of…
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Kalytera Therapeutics Will Meet with the FDA to Discuss Cannabidiol as a Possible Treatment For Acute GVHD
BrainStorm Cell Therapeutics Inc. has announced that it has secured its second U.S. manufacturing location for NurOwn®, an experimental drug currently under investigation for the treatment of amyotrophic lateral sclerosis…
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BrainStorm Has Secured a Second Manufacturing Facility for NurOwn, an Experimental Drug for ALS
Organisations are planning to develop new clinical practice guidelines for the diagnosis and management of von Willebrand disease. The original article can be read here, at PR Newswire (whose source…
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New Clinical Guidelines for Von Willebrand Disease Are Being Developed
A recently published study found that, out of the people studied, young cancer survivors were more likely to develop endocrine diseases than people who had no history of cancer. The…
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After Battling Cancer, Survivors Should Keep an Eye Out for Endocrine Disease, According to One Study
Researchers investigating the symptoms of testicular cancer have published their results in the British Journal of General Practise. Their study showed that testicular enlargement from a lump or swelling is…
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Researchers Are Working to Identify Common Signs of Testicular Cancer
Miracle Flights help children to fly to access medical treatment. They work to support people like Shelby, who was diagnosed with alveolar soft part sarcoma at the age of seventeen.…
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A Woman Diagnosed With Soft Part Sarcoma Received Treatment With the Help of Miracle Flights
A researcher from the University of Alabama at Birmingham is working on how to improve treatment for patients with status epilepticus. The original article can be read here, at UAB…
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A Researcher is Looking Into How Long Artificial Comas Should Last For Status Epilepticus Patients
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The final results from a study of the experimental drug tegsediTM (inotersen) for the treatment of patients with hereditary ATTR amyloidosis with polyneuropathy have been published. The original article can be…
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The Results of a Pivotal Study of Tegsedi For The Treatment of hATTR Amyloidosis With Polyneuropathy Have Been Published
Cleveland Clinic’s first every three-way kidney transplant has taken place. It involved six people in total: three donors and three recipients. The full article can be read here, at the…
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Cleveland Clinic Has Performed Their First Three-Way Kidney Transplant
The experimental drug olinciguat (IW-1701) has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of sickle cell disease. The full article can…
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ICYMI: Orphan Drug Designation Has Been Awarded to a Drug Being Developed to Treat Sickle Cell Disease
The Children’s Hospital Colorado has announced that it will part in a Phase 3 clinical trial for an experimental new cystic fibrosis therapy. The full article can be found at…
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A Hospital in Colorado Will Take Part in a Cystic Fibrosis Phase 3 Trial
A biotechnology company called Valneva is developing an experimental vaccine for Lyme disease. Valneva has said that it is making progress in the vaccine development. A Phase I trial has…
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A Vaccine Candidate For Lyme Disease Is Being Developed
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The Orphan Drug Designation awarded to the drug dovitinib for treating adenoid cystic carcinoma has been transferred from Novartis to Oncology Venture. The full article can be found here, at…
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The Orphan Drug Designation for the Drug Dovitinib Has Been Transferred to a New Company
A recent study has found that ‘clock’ genes that help to regulate the body’s circadian rhythm may be affected by spinal muscular atrophy. The original paper, which is published in…
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Researchers are Investigating the Link Between Circadian Rhythms and Spinal Muscular Atrophy
The US Food and Drug Administration (FDA) has approved an Investigational New Drug (IND) application for an experimental drug that is being researched for its use as a treatment for…
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A Study of an Experimental Drug for ANCA-Associated Vasculitis Will Go Ahead
A recent genetic study of early settlers in Iceland is hoped to shed light on certain genetic diseases, as well as the history of the population. The original articles can…
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Early Settlers in Iceland Had Surprisingly Different Genetics to the People Living There Now
Mepsevii (vestronidase alfa) is a treatment for Mucopolysaccharidosis VII (MPS VII). The Committee for Medicinal Products for Human Use (CHMP) has given a positive recommendation for the marketing authorisation of…
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A MPS VII Treatment Has Been Recommended For Authorisation in the EU
A new treatment for schizophrenia has been approved by the US Food and Drug Administration (FDA). Aristada Initio (aripiprazole lauroxil) for the initiation of Aristada (aripiprazole lauroxil) is now expected…
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A New Treatment Option For Schizophrenia Has Been Approved by the FDA
Researchers have found that taking nutritional supplements may affect the progression of genetically caused hearing loss. The full article can be read here, at Michigan Health Lab. Background to the…
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Nutritional Supplements May Affect Genetic Hearing Loss
The US Food and Drug Administration (FDA) is reviewing a supplemental New Drug Application (sNDA) for the drug Imbruvica (ibrutinib) in combination with rituximab (Rituxan) for treating Waldenström macroglobulinemia. The…
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A Supplemental New Drug Application Has Been Submitted For a Combination Therapy For Waldenström Macroglobulinemia
