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Thanksliving
We are entering the Thanksgiving season. I’ve been taught all my life that it shouldn’t be only a season, but an attitude year around. But how? By telling Mom thank…
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Thanksliving
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ICYMI: Does India’s Rare Disease Policy Only Exist on Paper?
According to a story from CheckOrphan, a wide array of experts are raising concerns about India's rare disease policy, which was first unveiled in May of 2017. Despite being hailed…
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ICYMI: Does India’s Rare Disease Policy Only Exist on Paper?
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Phase 1 Trial Shows Efficacy and Safety of New Glioma Treatment
Glioma is a type of cancerous tumor that originates in the brain. It has various types and the tumors range in aggressiveness. There are around 11,000 people just in the…
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Phase 1 Trial Shows Efficacy and Safety of New Glioma Treatment
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This Just In: India Never Implemented their Rare Disease Policy as Promised
We all got really excited last year when India implemented a new plan to benefit rare disease patients. It was called the National Rare Disease Policy. Basically, it put 12.86…
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This Just In: India Never Implemented their Rare Disease Policy as Promised
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A Clinical Trial for Gene Therapy is Underway for Muscular Dystrophy!
Limb-Girdle Muscular Dystrophy or LGMD, is a form of Muscular Dystrophy caused by a mutation which disrupts production of the protein beta-sarcoglycan. There are currently no treatment options for LGMD and…
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A Clinical Trial for Gene Therapy is Underway for Muscular Dystrophy!
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What do You do When You Can’t Get a Diagnosis?
According to a story from tonic.vice.com, Liz Aronin and Jamie Mills both thought that everything was going smoothly with their newborn son Quinn, but within a few months, it became…
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What do You do When You Can’t Get a Diagnosis?
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Study Shows the Most Effective Treatments for Polycythemia Vera are Under-prescribed
Polycythemia Vera or PV, is a rare blood cancer. It's caused by a mutation which results in the overproduction of red blood cells. Too many blood cells significantly increases your…
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Study Shows the Most Effective Treatments for Polycythemia Vera are Under-prescribed
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UK Cystic Fibrosis Patients are Protesting to get a Vital Drug Covered on the NHS
Cystic fibrosis patients in the UK are planning a protest on November 19th, 2018 with the goal of getting Orkambi, an important cystic fibrosis drug, covered on the NHS. This…
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UK Cystic Fibrosis Patients are Protesting to get a Vital Drug Covered on the NHS
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Ready to Donate to a Rare Patient on GoFundMe? Make Sure you are Paying for Something That Works
According to a story from ncbnewyork.com, a recent study found that $7 million on donations on crowdfunding sites for medical patients have gone towards treatments that are medically unsound and…
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Ready to Donate to a Rare Patient on GoFundMe? Make Sure you are Paying for Something That Works
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FDA Orphan Drug Designation for a New Neuroendocrine Tumor Treatment!
Neuroendocrine tumors are growths which form from neuroendocrine cells in various places throughout the body. They can occur in the gastrointestinal track, the pancreas, the lungs, the thymus, and other…
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FDA Orphan Drug Designation for a New Neuroendocrine Tumor Treatment!
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Study Shows Importance of Biobanks For Rare Disease Research
Biobanks store biological samples to help scientists research certain diseases. Registries collect information from patients and standardize it, but no samples are stored. Which of these is best for rare…
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Study Shows Importance of Biobanks For Rare Disease Research
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New Batten Disease Treatment Just Received Orphan Drug Designation!
Batten disease is a lysosomal storage disorder. It's neurodegenerative, meaning it results in the deterioration of neurons. One of the most common forms of this disease is caused by mutations in the…
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New Batten Disease Treatment Just Received Orphan Drug Designation!
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Previously Undiscovered Genetic Mutation Causes Intellectual Disability and Macrocephaly
According to a story from University at Buffalo, a recent discovery of a new genetic mutation continues to build on decades of research from Dr. Richard Gronostajski, a professor and biochemist…
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Previously Undiscovered Genetic Mutation Causes Intellectual Disability and Macrocephaly
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This Jewish Australian Teenager With Gaucher Disease Just Launched a Support Group for Patients
According to a story from the Australian Jewish News, a Jewish teen named Gidon Goodman was born with Gaucher disease. Recently, he started the first Gaucher disease patient support group…
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This Jewish Australian Teenager With Gaucher Disease Just Launched a Support Group for Patients
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Finding New Funding Approaches for Rare Disease Drug Development
According to a story from Raconteur, finding sufficient funding for the development of new therapies for rare diseases has always been a major challenge. Rare diseases are defined in the…
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Finding New Funding Approaches for Rare Disease Drug Development
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Participants in a Duchenne Muscular Dystrophy Clinical Trial Found out the Drug Wasn’t Working on Social Media
According to a story from nature.com, Amber Sapp's twelve year old son Garrett had been participating in a clinical trial that was testing a new potential treatment for his Duchenne…
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Participants in a Duchenne Muscular Dystrophy Clinical Trial Found out the Drug Wasn’t Working on Social Media
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This New Immunotherapy Could Allow for More Personalized Cancer Treatment
According to a story from UCI News, University of California Irvine scientists have developed an innovative immunotherapy that could screen patients in order to analyze the composition of their tumors…
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This New Immunotherapy Could Allow for More Personalized Cancer Treatment
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A Potential Treatment for Polio-Like Disease, Acute Flaccid Myelitis, Disappoints
According to a story from EurekAlert!, researchers have been scrambling to find a useful treatment for acute flaccid myelitis, a rare disease that has been appearing in unusual numbers in…
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A Potential Treatment for Polio-Like Disease, Acute Flaccid Myelitis, Disappoints
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Updates on PSP and CBD from the International Research Symposium
Often times it seems that research for the rarest of conditions is brushed aside. Primary attention is given to more common illnesses which affect more people. For instance, in the…
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Updates on PSP and CBD from the International Research Symposium
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Growing Older With Hemophilia: A Different Experience
As we age, we become more at risk for certain conditions. For instance, osteoporosis, atrial fibrillation, certain cancers, and heart disease, are more common in older individuals. These conditions pose…
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Growing Older With Hemophilia: A Different Experience
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These Families Facing Leigh Syndrome are Working for a Cure
According to a story from NRI Pulse, Kiran and Anju have been forced to watch over the past year as their daughter Aadya's physical and mental condition declined. After serious…
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These Families Facing Leigh Syndrome are Working for a Cure
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Scientists Identify Abnormal Immune Protein Activity as Trigger for Subcutaneous Panniculitis T Lymphoma
According to a story from Futurity, a recent study has identified a protein that plays a central role in the onset of a rare cancer called subcutaneous panniculitis T lymphoma…
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Scientists Identify Abnormal Immune Protein Activity as Trigger for Subcutaneous Panniculitis T Lymphoma
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A Real-Life Dallas Buyers Club? Cutting Long Lines and Buying Prescription Drugs Online
In the movie "Dallas Buyers Club" the main character, Ron Woodroof, contracts HIV/AIDs and starts getting drugs from an organization that imports medicine from Mexico. Similarly, many London patients are…
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A Real-Life Dallas Buyers Club? Cutting Long Lines and Buying Prescription Drugs Online
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A Device May Reduce the Uncertainty Around Shunt Failure For Patients With Hydrocephalus
In a recent news article, the Northwestern Medicine Feinberg School of Medicine has shared details about an experimental device that may have the potential to dramatically improve the treatment of…
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A Device May Reduce the Uncertainty Around Shunt Failure For Patients With Hydrocephalus
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Inborn Rare Disorders Like Pompe Disease Still Cause High Mortality
According to a story from Vietnam News, mother Huyen Trang has seen two of her children face death's door within mere months of being born. Her first child died at…
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Inborn Rare Disorders Like Pompe Disease Still Cause High Mortality
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