As originally reported in ArcaMax, when you get a diagnosis for a terminal rare disease, a wave of emotions crashes over life. Brian Wallach emerged clutching 'hope.' In 2017, Brian,…
A family is grieving for their daughter and taking it one day at a time in the face of a terminal diagnosis. Kelly and Andrew Mills became pregnant with…
Spinal muscular atrophy (SMA) currently has two approved treatment options. Just 3.5 years ago this number was zero and in another year or so, researchers are hoping to increase it to…
A study published on February 3rd, 2020 in the scientific journal Nature Research is highlighting the capability of a three part combination treatment for use in multiple myeloma, a rare and…
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According to a report in Myeloma Research News, the European Commission has recently granted authorization to market bortezomib, dexamethasone and thalidomide (collectively known as VTd) plus daratumumab to treat…
According to a story from Salon, the Quality Adjusted Life Year (QALY) is a statistical measure that is used in order to calculate the value of a certain medication. First…
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A recent press release by the global biopharmaceutical research company, AbbVie, reported a positive opinion by CHMP granted to VENCLYXTO® combined with obinutuzumab to treat chronic lymphocytic leukemia (CLL). A positive opinion by…
Zynerba Pharmaceuticals has recently announced that they have finished the patient screening for their clinical trial of Zygel, a treatment for Fragile X syndrome. Zygel is a CBD gel that…
When Jennifer Payne was born in 1973, she was diagnosed with a rare disease: phenylketonuria (PKU). This genetic disorder is typically screened for at birth. This early screening process is…
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…
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…
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…
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 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 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…
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…
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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