The International Pain Foundation’s All in 1 iPain Summit
The All in 1 iPain Summit Los Angeles, CA #GetOnTrack This conference is dedicated to patients that are dealing with chronic, long term pain. This event will inform attendees about…
Kisaco Research’s Rare Disease Diagnostics Summit
Rare Disease Diagnostics Summit "Reducing the Diagnostic Odyssey to Improve Patient Outcomes" Boston, Massachusetts The diagnostic component of genetic rare diseases must be addressed. More than 350 million people are…
The Chinese Organization for Rare Disorders’ 8th China Rare Disease Summit
The Eighth China Rare Disease Summit The China Rare Disease Summit is the largest rare disease focused event held in China. A variety of rare disease stakeholders from across the…
Global Genes’ RARE Patient Advocacy Summit
The Global Genes RARE Patient Advocacy Summit This conference will be a convergence of a variety of rare disease stakeholders, such as rare disease patients, world renowned experts, and caregivers.…
Everylife Foundation for Rare Diseases 11th Annual Scientific Workshop
The 11th Annual Scientific Workshop Science of Small Trials in the Age of Biological Plausability The 11th Annual Scientific Workshop will be a convergence of key regulatory officials, researchers, and…
The National Organization for Rare Disorders 2019 Patient & Family Forum
The 2019 Living Rare, Living Stronger NORD Patient & Family Forum Featuring The 2019 Rare Impact Awards As published on NORD's website, the 2019 Living Rare, Living Stronger NORD Patient…
The National Organization for Rare Disorders’ Living Rare, Living Stronger NORD Patient & Family Forum
The 2019 Living Rare, Living Stronger NORD Patient & Family Forum featuring the Rare Impact Awards 2019 This rare disease patient forum brings together health professionals, physicians, patients, and medical…
The Oley Foundation’s Oley Foundation and University of Illinois at Chicago Conference
The 2019 Oley Foundation and University of Illinois at Chicago Annual Conference The Essentials of Home Parenteral and Enteral Nutrition: What Consumers and Clinicians Need to Know Are you a…
Issues with Quality Assurance of Generic Drugs and How We Can Improve the Process
What are Generic Drugs? Generic drugs are any medications "equivalent" to brand name pharmaceuticals. These drugs are much cheaper than the brand name medications and the United States Food and…
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Duchenne Muscular Dystrophy and The Heart: Five Facts You Should Know
According to a story from blog.cincinnatichildrens.org, a diagnosis of Duchenne muscular dystrophy is always an alarming piece of news; learning that your child has a progressive muscle atrophy disorder that…
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Doctors Say Border Patrol is Confiscating Life-Saving Medications from Detained Migrants
According to a publication from Yahoo News, five doctors interviewed by the media organization have reported meeting with a number of migrants detained by United States Customs and Border Protection…
Researchers Find Gut Bacteria that Can Change Type A Blood to Type O
Blood Donations For many people living with a rare disease such as Beta thalassemia, Hemophilia, Aplastic Anemia, Myelodysplastic syndromes, Acute Promyelocytic leukemia, and others, blood transfusions are not uncommon. For…
12-Year-Old with Idiopathic Thrombocytopenic Purpura Creates Nonprofit to Make Transfusions Easier for Kids
Ella Casano is a 12-year-old living with Idiopathic Thrombocytopenic Purpura (ITP) who had a brilliant idea about how she could help other children living with rare diseases like her own.…
Attend the Rare Disease Diagnostics Summit in Boston This Fall
Mark your calendars! The Rare Disease Diagnostics Summit is being held in Boston, MA on September 24th and 24th, 2019. This incredible event will bring together pharmaceutical companies, biotechs, diagnostics,…
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Spirit Lake Woman Faces Several Surgeries to Treat Chiari Malformation
After years of having multiple physical complications and problems, a young woman in Spirit Lake found medical answers in 2016. In that year, Jessica Brown was diagnosed with Chiari…
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Alnylam Pharmaceuticals Submits New Drug Application for Experimental Acute Hepatic Porphyria Drug
According to a press release from Alnylam Pharmaceuticals, the Massachusetts-based biopharmaceutical company has completed the submission of its New Drug Application (NDA) for givosiran — its experimental acute hepatic porphyria…
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New Discoveries Reveal Further Connections Between the Gut Microbiome, Human Health, and Rare Diseases
According to a story from bioengineer.org, a team of researchers associated with Oregon State University are making further headway in understanding the connection between our health and the bacteria that…
Researchers Uncover New Approach to Stem Cell Therapy which Could Effectively Treat Multiple Sclerosis
Stem Cell Therapies While stem cell therapies hold a great deal of promise for many rare diseases, there is still a lot that we don't understand. Experimental stem cell therapies…
Women with Breast Cancer Risk Must Weigh the Unknowns of Certain Types of MRIs
Meredith Resnick's breast cancer risk is 42%. This risk is made up of her higher breast density (denser breasts are associated with increased risk), Ashkenazi Jewish heritage, a family history…
Researchers Discover Way to Modify Rheumatoid Arthritis Drugs to Reduce Immune Suppression
"A new way to improve the efficacy of a drug taken by millions of patients throughout the world." Background Adalimumab (also known as Humira) and infliximab (also known as Remicade)…
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Experimental Spinocerebellar Ataxia Treatment Earns Orphan Drug Designation
According to a story from Bloomberg, the company Cadent Therapeutics recently announced that the company's experimental product candidate CAD-1883 has earned Orphan Drug Designation from the US Food and Drug…
Phase 2 Clinical Trial for Microvillus Inclusion Disease Approved to Begin in Turkey
MVID Microvillus inclusion disease (MVID) is a rare, gastrointestinal disease which can cause impaired development in children. It is characterized by severe diarrhea which leads to dehydration and malnutrition. MVID…
Editor’s Choice: Shady Stem Cells, Anti-Virals and Young Adults Fighting Cancer
Happy Thursday! Today, we're highlighting four articles on rare disease news. First, we have a story on a lawsuit against controversial stem cell treatments. Next, we have an article on…
Potential Stargardt Disease Therapy Receives Orphan Designation by EMA
Stargardt Disease Stargardt disease is a rare condition that causes vision loss due to the buildup of vitamin A byproducts in the retina. The byproduct causing the largest buildup is…
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FDA Wins Major Lawsuit Against Shady Stem Cell “Therapy” Company, U.S. Stem Cell
According to a publication from The Portland Press Herald, Judge Ursula Ungaro, a senior District Judge in the Southern District of Flordia, recently ruled in favor of the United States'…
