As originally reported in Cambridge Independent, inflammatory bowel diseases are about to get a boost in research after Health Data Research UK has awarded a £5 million grant to a…
With advocacy organizations gaining traction, sometimes patients with rare diseases may have a handful of options to choose from. Some patients may be quick to join patient advocacy groups…
The Bad News Unfortunately, it has recently been announced that an experimental treatment for facioscapulohumeral muscular dystrophy (FSHD) will not be continuing on in clinical trials. This is because the…
Innovation and Collaboration Macular telangiectasia type 2 (MacTel) is a rare eye disease that causes progressive vision loss as patients age. Paul Bernstein is a physician at the John A.…
According to a story from Fabry Disease News, the first patient in a clinical trial testing the investigational gene therapy FLT190 has reportedly been dosed. FLT190 is being developed as…
According to a story from finanznachrichten.de, the latest data from three phase 3 studies is giving further confirmation to the capability of the recently approved gene therapy Zolgensma to have…
According to a story from Spectrum News a recent study revealed two potential therapies that could treat symptoms associated with Rett syndrome, a rare disorder. The study involved the creation…
According to a story from ANCA Vasculitis News, a recent study has found that a low-dose regimen of glucocorticoids that explicitly exclude the drug methyprednisolone can be a useful therapy…
According to a story from Scleroderma News, the results of a recent mouse study could have significant implications for the future of scleroderma treatment. In the study, scientists used a…
According to a recent press release from the Pennsylvania-based Marinus Pharmaceuticals, the Company is reporting positive results of its phase 2 study of "ganaxolone," an investigational GABAA modulator for patients…
According to a story from Healio, a recent study has determined that older patients with multiple myeloma, a rare cancer that affects plasma cells, routinely under-utilize supportive care resources as…
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Pamela Smith, age 67, had run out of options when her melanoma metastasized and became inoperable. According to a recent article in MedicalXpress, in January 2014, Pamela joined the…
According to a publication from EurekAlert, a study recently published in the medical journal Nature Genetics suggests how certain immune cells express genetic activity similar to that of variations of the…
A study record from clinicaltrials.gov is investigating the appearance of lung problems in patients with disorders affecting connective tissue. While prior research has documented the appearance of interstitial lung disease…
According to a story from Juvenile Arthritis News, a recent study has found that the drug leflunomide (marketed as Arava) may be a viable treatment for juvenile idiopathic arthritis patients,…
According to a story from BioSpace, a recent study of cancer in children is emphasizing the fact that genetic testing is an essential component of cancer treatment, providing critical information…
According to a story from PharmaTimes, the gene therapy company Bluebird bio has released updated results from a clinical study of its Lenti-D gene therapy as a treatment for patients…
According to a story from Pompe Disease News, a recent study has concluded the polymorphisms of the ACE gene (which are genetic variations that occur naturally) do not have an…
According to a story from Pompe Disease News, a recent study has found that delays in diagnosis are closely connected to whether someone displaying symptoms of Pompe disease is referred…
According to a story from Parkinson's News Today, a phase 2 clinical trial that will test a gene therapy for advanced Parkinson's disease has begun recruiting patients. The experimental gene…
According to a story from sectorpublishingintelligence.co.uk, the drug company Ovid Therapeutics Inc. has recently announced that the early data from its phase 2 clinical trial is mostly encouraging. This clinical…
As originally reported in Biobuzz, the story of rare disease treatment took a turn in the age of modern technology. Rare diseases have traditionally been inaccessible to the naked eye,…
According to a story from Angioedema News, a recent study demonstrated the effectiveness of the medication Haegarda in reducing the number of swelling attacks experienced by hereditary angioedema patients. Preventative…
According to a story from Myeloma Crowd Crowdcare Foundation, the very first Plasma Cell Disorder Congress recently took place in Charlotte, NC from August 10-11, 2019. The event primary focused…
According to a story from globenewswire.com, the gene and cell therapy company Abeona Therapeutics Inc. recently announced that has received a letter from the US Food and Drug Administration (FDA)…
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