By Natalie Homan from In The Cloud Copy Rochelle was a 28-year-old mom of two when she went to sleep one night feeling perfectly normal. A few hours later, she…
Chive Charities is a 501(c)3 non-profit organization committed to providing patient advocacy and support. They have raised money for a young girl with a mutated CAMK2B gene to receive intensive physical therapy at…
by Danielle Bradshaw from In The Cloud Copy A year-long study has shown that Tracleer greatly reduces the skin-related symptoms of scleroderma. The results also showed that although it helped…
May is recognized annually as Cystic Fibrosis (CF) Awareness Month. Each year, this rare disease community comes together in order to help spread awareness about cystic fibrosis among the general…
Researchers at the University of Houston have been investigating lupus nephritis and discovered that there are race-specific urinary biomarkers. Hugh Roy and his team have found that there are specific…
Bronchiectasis News covers a potentially beneficial treatment for patients with bronchiectasis: bubble-positive expiratory pressure, or Bubble-PEP. These devices help clear mucus from the lungs. Although other forms of airway clearance…
According to a story from Xconomy, the drug company PTC Therapeutics has come to an agreement that will allow it to acquire Censa Pharmaceuticals and its lead product candidate. This…
A team of researchers recently clarified how cellular mechanisms work in the treatment of Krabbe disease. Previously, researchers believed that bone marrow transplants assisted in providing positive patient outcomes through…
ProMIS Neurosciences is a biotechnology company that works specifically with antibody treatments, and they have recently developed intrabodies for the treatment of amyotrophic lateral sclerosis (ALS). This same therapy has…
It is acknowledged in the medical community that clumps of misfolded tau proteins and amyloid-β are markers of Alzheimer’s disease. The evidence presented in recent article in Nature, shows that…
According to UIC Today, Jessica Lechuga became immersed within the rare disease community when she was still a teenager. That's because, at age 17, Jessica gave birth to her daughter…
According to WMC5, a research study out of Cleveland, OH, has been testing the impact of robotic exoskeletons on movement for patients with multiple sclerosis. For Kathy Miska, this is…
According to a recent article in BioMed Central, an international task force formed in 2018 created guidelines for the treatment of juvenile idiopathic arthritis (JIA). Treatment targets were identified…
Patients with hemophilia may experience sudden bleeding, joint pain and inflammation, and deep bruising. But according to Hemophilia News, two proteins may be the cause. Researchers found that patients…
Continued From Part Two Written by Payel Bhattacharya I hope for aid from the government to cover the high cost of subsequent medicines including anti-rejection medicines, radiological and lab…
Kubota Vision, a subsidiary of Kubota Pharmaceutical Holdings, has recently finished enrollment in the third phase of their emixustat hydrochloride (emixustat) trial. Emixustat hydrochloride is a treatment for Stargardt disease…
Merck and AstraZeneca recently announced that Lynparza improved overall survival in the lives of males with advanced prostate cancer who had been treated previously. The drug out-performed two other antiandrogen…
Of course, parents are not used to taking on the role of educator. Many are still working full time jobs at home. That, in addition to the normal household chores,…
According to Angelman Syndrome News, researchers used rat models of Angelman syndrome to test gene editing as a potential therapy. They found that CRISPR technology deleted the defective UBE3A gene. However, rats…
As COVID-19 continues to spread across the globe, more and more people are wondering how this virus might affect them. This is especially true for patients with rare diseases,…
Continued From Part One written by Payel Bhattacharya Pharmaceutical science has medications to control the pain. I was initially put on medicine but it mostly didn’t contain the pain. I…
Saying that COVID-19 has impacted the world might just be an understatement. In fact, COVID-19 changed the way we interact with each other, businesses, and the world around us. But how…
By Payel Bhattacharya, living with von Hippel-Lindau syndrome I, Miss Payel Bhattacharya, was an unusual child. I was never concerned with petty things, the trivia, the dregs in life. My…
Recently, Pfizer's Dr. Ian Winburn sat down with Europabio for an interview in honor of World Hemophilia Day, which just occurred on April 17. In his discussion, Dr. Winburn…
Phase III of the CheckMate -9ER trial has reached its primary endpoint of progression free survival in those with advanced or metastatic renal cell carcinoma. This study was evaluating CABOMETYX…
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