According to a story from Essex Live, Lily Wythe is a fourteen year old girl who was recently diagnosed with a rare form of brain cancer called diffuse intrinsic pontine…
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According to an article in the Alzheimer’s publication Being Patient, participants who had been enrolled in the discontinued phase three trials of aducanumab will start receiving the drug as…
Asfotase alfa is an enzyme replacement therapy meant to directly address the cause of hypophosphatasia. While more research is necessary, it has been shown to decrease the mortality rate and…
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According to a January 2020 release by Business Wire, the USFDA has awarded accelerated approval of Epizyme’s TAZVERIK™ in the treatment of advanced epithelioid sarcoma (ES). The approval was…
PTC Therapeutics is a pharmaceutical company that has been working with Translarna, a medication intended for the treatment of Duchenne muscular dystrophy (DMD). They analyzed this treatment and presented the…
Orchard Therapeutics is investigating their gene therapy, OTL-102, for the treatment of X-linked chronic granulomatous disease (X-CGD). This treatment has recently received the Orphan Drug designation from the FDA, which…
According to a story from The ASCO Post, the promising results from a phase II clinical trial conducted last year suggests that CAR T-cell therapy could be an effective treatment…
Happy Thursday! This week, we're highlighting a story about a story about Karina, an EDS patient and advocate. Next, we have a story about a legal controversy that could affect…
According to a story from USA Today, 61 year old Jerry Devries is currently the oldest living patient with a very rare disease: Proteus syndrome. An unusual disease with only…
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Janssen R&D, a division of Johnson & Johnson (J&J), has partnered with the UK Biobank and others according to a recent news release by 3BL Media. It is noteworthy…
According to a story from Pulmonary Advisor, the results of a recent analysis led researchers to conclude that systemic anticoagulation therapy can be beneficial for patients with idiopathic pulmonary arterial…
Hereditary Angioedema Hereditary angioedema (HAE) is a rare disease which causes swelling episodes throughout the body, including the airways, gastrointestinal system, and extremities. Most commonly, HAE is caused by a…
According to a story from Angioedema News, a team of researchers and scientists have successfully developed a tool that is able to measure disease activity in hereditary angioedema that is…
According to a story from the Stamford Advocate, rock legend Ozzy Osbourne is making plans to go on tour again next fall. This is despite the fact that he has…
According to a story from The ASCO Post, the drug cabozantinib (marketed as Cometriq) was first approved by the US Food and Drug Administration (FDA) as a treatment for metastatic…
According to a story from the ASCO Post, the US Food and Drug Administration (FDA) first approved the drug pembrolizumab as a treatment for advanced esophageal squamous cell carcinoma on…
According to a story from newswiretoday.com, the biopharmaceutical group Ipsen has recently announced its decision to halt its dosing of patients in its phase 3 clinical trial and its phase…
As originally reported in BioSpectrum; the World Health Organization (WHO) has just approved the disease code for CDKL5 Deficiency Disorder (CDD), adding it to the list used worldwide for the…
According to a story from Malaysian Tribune, the pharmaceutical company Khondrion has just announced that the first patients have been dosed in its phase IIb clinical trial. This trial is…
'What you can't see can't hurt you.' It's a classic and well-intentioned phrase, but we know it to be quite untrue; especially if you ask any one of the millions…
According to a story from The ASCO Post, the drug dasatinib (marketed as Sprycel), was officially approved as a treatment for chronic myeloid leukemia by the US Food and Drug…
As originally reported in Cystic Fibrosis Life Foundation, Martha has spent her 24 years of life with cystic fibrosis. She was born with the rare disease which means for a…
Rubius Therapeutics is a biopharmaceutical company that focuses on cellular medicines. They have recently dosed the first patient in Phase 1b of their clinical trial of RTX-134. This therapy is…
Many diseases are caused by mutations in the DNA. In order to understand these diseases, it is necessary to understand the human genome. Janssen Research & Development has been working…
Ipsen is a pharmaceutical company based out of Paris. Sadly, they have just announced that they have pressed pause on not one but two studies for fibrodysplasia ossificans progressiva (FOP). FOP…
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