Neurogene, which works to provide treatments for rare neurological disorders, has recently joined with Behind the Seizure. Behind the Seizure was established by two other companies, BioMarin and Invitae, with…
Methylmalonic Acidemia Methylmalonic acidemia (MMA) is a rare disease affecting the metabolic system. 60% of all cases are due to a deficiency in MUT, a mitochondrial enzyme, which causes acids to…
According to a story from gurufocus.com, the pharmaceutical company Zogenix, Inc., has recently announced top-line results from its phase 3 clinical trial. This trial is testing FINTEPLA® as a treatment…
An article in CURE® describes two extremely rare types of tumors: paraganglioma and pheochromocytoma. One in three thousand individuals may have either or both of these tumors. The symptoms accompanying…
As originally reported in Mayo Clinic, Dr. Veronique Belzil realized she wanted to leave her job as a psychologist to find a cure for amyotrophic lateral sclerosis when she saw…
According to a study published in the scientific journal Neurology, the survival rates of patients with Lambert-Eaton myasthenic syndrome (LEMS) were compared to that of the general population as well as…
According to a story from mmm-online.com, a recent infographic highlights the challenges that rare disease patients worldwide still face when it comes to getting the treatment that they so desperately…
NeuroSense Therapeutics, a biotechnology company, is in the process of developing PrimeC. PrimeC was created for the treatment of amyotrophic lateral sclerosis (ALS), and it is meant to slow or…
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On February 3, 2020, twenty-seven newly installed flags of the European Union stood in the gleaming lobby of the new European Medicines Agency (EMA) headquarters in Amsterdam. The Charcot-Marie-Tooth…
According to a story from Charcot-Marie-Tooth News, the Muscular Dystrophy Association (MDA) may at first seem like a group that is solely focused on helping muscular dystrophy patients, but its…
Having a rare disease means you could have one of any of 5,000-7,000 distinct disorders, each with their own quirks. Just how many there are though remains unclear. As originally…
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Scientists in four continents worked for over a decade on a massive project that has resulted in the most detailed picture of cancer ever produced. Researchers now have a huge…
The National Center for Advancing Translational Sciences (NCATS) of the National Institutes of Health (NIH) and the Center for Biologics Evaluation and Research (CBER) of the Food and Drug…
As originally reported in Financial Buzz, Rett Syndrome has received promising news: a drug undergoing review, ANAVEX®2-73 (blarcamesine), has received fast track designation, meaning patients may be able to receive…
According to a story from curetoday.com, Dr. Tara Seery is an investigator in a phase 3 trial called PROOF, and she has just one goal: finding better treatments for cholangiocarcinoma,…
NeuroQWERTY is a device created by nQ Medical that is meant to monitor brain health and the progression of the disease in those with Parkinson's. It has recently been granted…
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…
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…
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