According to a story from globenewswire.com, the biopharmaceutical company Inventiva is scheduled to present the findings from a recent phase IIa clinical trial at the upcoming 16th Annual WorldSymposium™ which is…
Losmapimod is an experimental treatment for facioscapulohumeral muscular dystrophy (FSHD), and it recently received the Orphan Drug designation from the FDA. Not only did it receive this designation, but its…
As originally reported in StatNews, Mark Zuckerberg, founder of Facebook, along with his wife, Dr. Priscilla Chan, a former pediatrician and now head of the Chan Zuckerberg Initiative, made an…
According to a story from BioSpace, the biopharmaceutical company Insmed Inc. has recently announced favorable results from its phase II clinical trial. The clinical trial is testing the company's investigational…
According to a story from Chronicle Live, 36 year old Donna Campbell was found unresponsive in her hospital bed on June 22nd, 2019. She was officially pronounced dead the following…
According to a story from Medical Xpress, a new technological approach developed at the University of Texas Health Science Center at Houston will allow doctors to distinguish between Parkinson's disease…
According to a story from globenewswire.com, the biotechnology company Emerald Health Pharmaceuticals Inc., has announced recently that its experimental drug EHP-102 has earned Orphan Drug designation from the European Medicines…
Front Line Genomics has recently interviewed Dr. Michelle Krishnan, who is the Translational Medicine Leader in Rare Diseases at Roche. She focuses on rare neurodevelopmental disorders, in which she works…
VistaGen Therapeutics has recently been cleared by the FDA to begin the second phase of their study of AV-101, which is intended for the treatment of dyskinesia in those affected…
According to a story from Financial Buzz, the biopharmaceutical company miRagen Therapeutics has recently announced the release of new data from its phase I clinical study. This trial is testing…
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…
In another article I wrote for Patient Worthy, I talked about the emotional pain of “victim mentality” when faced with a health or other life challenge. In this article, I…
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…
Dr. Halima Moncreiffe is an assistant professor at Cincinnati Children's Hospital Medical Center, and she focuses on autoimmune diseases. She recently participated in an interview with Front Line Genomics, in…
Phase 3 Trial A Phase 3 trial run by Pharnext examining the safety and the efficacy of an investigative therapy called PXT3003 began in December of 2015. It enrolled 323…
Lisa Christopher-Stine is the Director of the Myositis Center at Johns Hopkins. She recently shared her biggest tips for patient care and some updates from the field, hoping to change…
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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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The Halcyon machine holds an imaging mechanism that produces a high-quality CAT scan plus it houses a treatment unit that delivers radiation. According to a recent news release, approximately twenty-four…
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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…
The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two…
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…
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