In Matthew Hutson’s contribution to Medium’s “Future Human” series, he interviews George Church. Church, the Harvard geneticist, molecular engineer, and chemist has been hugely influential in the understanding of DNA.…
According to a story from PharmaTimes, the pulmonary arterial hypertension drug selexipag, developed by Actelion, was unable to make the cut to obtain coverage on the NHS in England. The…
Recently in Charcot‐Marie‐Tooth news, progress has been made in identifying better testing to use as outcome measures for people living with Charcot-Marie-Tooth disease type 1A. A study called “Outcome measures…
According to a story from Deseret News, after she was diagnosed with multiple myeloma, Jenny Ahlstrom almost immediately had to make crucial decisions regarding her treatment: decisions that could mean…
Zynerba Pharmaceuticals, Inc. has announced their plans to begin a clinical trial of Cannabidiol in children and teenagers who have Fragile X syndrome. The study, called CONNECT-FX, is expected to…
This is a continuation of An Interview with the Unstoppable Helena Baker. We had just learned about the multiple decades it took for her to receive her diagnosis of Fibula…
According to a July 9 report by the Muscular Dystrophy Association (MDA), the MDA awarded a large grant to researchers studying Charcot-Marie-Tooth disease. The grant, totaling over $1,000,000, was awarded…
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…
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…
Danielle and Jonathan Foltz live on the New England coast with their three children. In an CNN article, Wayne Drash documents their meteoric fall from a mission family with big plans…
According to a June 20th report by the National Hemophila Foundation (NHF), the NHF along with several other prominent organizations in the bleeding disease community will be joining forces to…
According to a story from PBS, the FDA has been approving new treatments at a noticeably faster rate than it has in the past. As a result, it appears that…
BuzzFeed News reported a few weeks ago about the tragic story of 16-year-old Sara Manitoski who died of Toxic Shock Syndrome: a rare condition with a very common source. Toxic shock syndrome…
Happy Friday, everyone! We hope everyone's drinking water, wearing sunscreen, and taking a little time to relax this summer. As we get ready for the weekend, we've gathered four recent…
According to a story from EurekAlert!, researchers have found that the drug Diamox, which is used to treat a variety of illnesses and conditions such as altitude sickness, glaucoma, epilepsy,…
According to a story from bioportfolio.com, two companies, Ionis Pharmaceuticals, Inc., and its affiliate Akcea Pharmaceuticals, recently announced that its product inotersen (to be marketed as TEGSEDI) has gained approval…
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…
According to a story from the Washington Times-Reporter, Michelle Marcinak's three sons-- John Henry, Leo, and Rafe-- were all diagnosed with the rare gastric condition eosinophilic esophagitis. Early symptoms of…
According to a story from BioSpace, the biopharmaceutical company Acceleron Pharma, which develops TGF-beta based treatments for rare and serious illnesses, recently announced that the U.S. Food and Drug Administration…
According to a story from ScienceDaily, a recent analysis has been conducted in order to investigate patient outcomes for children with the rare skin condition Stevens-Johnson syndrome, as well its…
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…
According to a story from NPR, plastic straw bans have been gaining traction across the U.S. One of the most shocking examples of planetary degradation as the result of human…
According to a story from the Globe and Mail, Michelle Quinlan was overwhelmed when her child Avery was diagnosed with Coffin-Siris syndrome, a rare genetic disease that can cause intellectual…
According to a story from Global News, direct-to-consumer genetic tests are becoming increasingly popular. These tests can be used to learn new info about your ancient ancestors, closer relatives, and,…
New pig models of Huntington’s disease have been developed, and they are expected to have several benefits compared to existing rodent models of the condition. Read the source article here…
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