According to a story from outsourcing-pharma.com, the US Food and Drug Administration (FDA) has recently started a new initiative with the opening of its Compounding Quality Center of Excellence. Janet…
Orphazyme, a biopharmaceutical company, has recently released the data from their study of arimoclomol. This product is meant for the treatment of Niemann-Pick disease type C (NPC). Orphazyme specializes…
Herantis Pharma has been conducting a study of their treatment for Parkinson's disease, which is cerebral dopamine neurotrophic factor (CDNF). Phase 1/2 has been completed, and an extension phase…
According to an article in BioSpace, the FDA recently approved the PARP inhibitor Lynparza that was developed by Merck and AstraZeneca to be used as maintenance therapy for pancreatic…
According to a story from Medgadget, a team of scientists from Columbia University have been working on a robotic device that could assist patients with spinal cord injuries and impairments…
January is Cervical Health Awareness Month! The New Year holiday is all about taking stock of one's self and making efforts to improve or set goals for a better you.…
Doctor Daniel Kastner, director of NHGRI, recently uncovered a new rare disease along with his research team. It’s called CRIA syndrome. This discovery has been published in Nature. CRIA CRIA…
A New Working Group A team of healthcare professionals from the Institute for Quality and Efficacy in Health Care, the Drug Commission of the German Medical Association, and the Charité…
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Susan Winn’s family history of ovarian and breast cancer can be traced back to the 1860s. Susan’s adult daughters, Kathryn, Bridget and Maureen, were recently interviewed with CURE® (Cancer Updates Research…
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An article published earlier this year by Fierce Biotech outlines an approach that was developed by researchers at the Fred Hutchinson Cancer Research Center. Using CRISPR-Cas9’s gene-editing method the researchers were…
Happy New Years! We're back with a batch of new article! This week, we're highlighting four stories: an interview on 20 years with dystonia, a young woman stuck in a…
As originally reported in EurekAlert, creating effective rare disease pharmaceuticals is difficult. Yes, because it is expensive, there’s a small population to use in trials, and there’s limited research. Sometimes…
The human spirit can be a remarkable force. Robb Freed cycled thousands of miles by himself to raise awareness for epidermolysis bullosa (EB) -- a rare illness that took the…
The annual Code Talker award was presented during an engaging ceremony at the National Society of Genetic Counselors’ annual conference. Every year, patients submit essays nominating their genetic counselors for…
Treatment for ulcerative colitis differs based on the individual. Where the inflammation occurs, the severity, and the preference of the patient can all influence the treatment. For those with moderate…
As originally reported by the Oklahoma Medical Research Foundation; Tonja Martin was running through her retirement savings for her medical care. She spent $3,400 every month to cover treatment for…
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According to a recent article in Euronews, this year one hundred children who were diagnosed with spinal muscular atrophy (SMA) will receive Zolgensma, the world’s highest-priced drug, at no cost.…
TrialJectory is an organization working to match bladder, breast, colorectal, melanoma, myelodysplastic syndromes (MDS), and other cancer patients with the clinical trial that is right for them. They have successfully raised…
GlaxoSmithKline (GSK) has recently completed and released the results of Phase 3 of its study of benlysta. This drug is made for the treatment of lupus nephritis. Their BLISS-LN…
Who is most affected when we talk about creating solutions for rare diseases? Those who live with them and their families. Patient communities know what they need and feel the…
Savara Pharmaceuticals has recently interviewed Dr. Tisha Wang, who is a pulmonologist and critical care physician at UCLA. Dr. Wang is also an advocate for autoimmune pulmonary alveolar proteinosis…
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According to recent articles in Runner’s World Magazine, having a diagnosis of multiple sclerosis has not stopped Cheryl Hile, Jennifer Lee or Melissa Ossanna. Each woman is fighting the symptoms…
This is NSGC 2019 Conference Highlights Part 2, click here for part 1. An early morning session on the final day of the conference featured a personal story shared by…
Written by: Paul Pavao The most important thing I can tell you is that Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) can be survived. As of 2019 survival requires a bone…
This interview was originally published on the AMADYS website I was recently interviewed by Matthieu Creson for AMADYS to share my experience living with dystonia for nearly 20 years, as…
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