Study Indicates Safety of New Potential Treatment for Swallowing Dysfunction in Kennedy Disease
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Study Indicates Safety of New Potential Treatment for Swallowing Dysfunction in Kennedy Disease

Kennedy disease or, Spinal and Bulbar Muscular Atrophy (SBMA) is a form of Spinal Muscular Atrophy (SMA). SBMA causes muscle weakness throughout the limbs which results in impaired mobility (typical…

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Bioscience Companies in Ohio Take Aim at Rare Diseases
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Bioscience Companies in Ohio Take Aim at Rare Diseases

According to a story from BioPortfolio, Rare Disease Day, which took place on February 28th, 2019, serves as a time of reflection on the progress that has been made in…

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The Major Ethical Dilemma Behind Rare Disease Drug Costs and a Bioethicist’s Solution

Avraham Steinberg recently spoke at RARE2019, the 2nd International Congress on Advanced Treatments in Rare Diseases. It was held on March 4th and 5th in Vienna, Austria. His talk focused…

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New Treatment for Familial Chylomicronemia Syndrome is Close to Approval in the EU

Familial Chylomicronemia Syndrome Familial chylomicronemia syndrome (FCS) is a condition that is considered ultra-rare. It's caused when the enzyme called lipoprotein lipase (LPL) is impaired. It is can result in…

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Ireland Lags Behind the Rest of Europe in Approving Rare Disease Drugs
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Ireland Lags Behind the Rest of Europe in Approving Rare Disease Drugs

According to a story from The Irish Times, a recent report issued by the Irish Pharmaceutical Healthcare Association (IPHA) indicates that Ireland trails many other European countries in approving new…

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FDA Releases New Info About How it Could Regulate Gene Therapy

According to a story from finance.yahoo.com, the US Food and Drug Administration (FDA) has recently released new guidelines that are related to how the agency will regulate and manage the…

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Passage Bio Receives New Funding to Advance 5 Gene Therapy Projects
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Passage Bio Receives New Funding to Advance 5 Gene Therapy Projects

Passsage Bio Passage Bio has just announced that they've received 115.5 million dollars to help advance their development of gene therapies. They will begin by using the funds to further…

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Choroideremia Can Lead to Blindness – But Gene Therapy Might Be Able to Halt It
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Choroideremia Can Lead to Blindness – But Gene Therapy Might Be Able to Halt It

An article recently published by the Guardian shared the stories of three families affected by recent breakthroughs in gene therapy treatments. What is Gene Therapy? Gene therapy is an increasingly…

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Her Brother Participated in Trials for Vital Spinal Muscular Atrophy Drug, and Now She’s Denied Treatment Access

According to a story from mirror.co.uk, a five-month-old girl named Maryam Malji was diagnosed with spinal muscular atrophy, a progressive, degenerative disease that is ultimately fatal without treatment. Her brother…

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You Can Provide Suggestions for the FDA’s Revision of their Rare Disease Research Guidelines

Time for Revision Like many things in this world, research for rare diseases such as Spinal Muscular Atrophy (SMA), Multiple Sclerosis, and Duchenne Muscular Dystrophy (DMD) has evolved substantially in recent years. This evolution…

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Arkansas Bill Aims to Add Spinal Muscular Atrophy to The State’s Newborn Screenings
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Arkansas Bill Aims to Add Spinal Muscular Atrophy to The State’s Newborn Screenings

According to a story from arkansasonline.com, Rep. Julie Mayberry R-Hensley of the Arkansas General Assembly is sponsoring House Bill 1074. This legislation proposes adding the rare and potentially fatal genetic…

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30 Million Euros Allocated to Fund Precision Medicine Research Projects for Neurodegenerative Diseases

Precision mNedicine is the latest craze in rare disease research. Thankfully for patients, it is completely, 100% centered on improving the quality of their care. Unlike most typical healthcare practices,…

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Massachusetts Proposes Installment Plan to Cover Cost of Gene Therapy for Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is a genetic condition which affects between 10,000 and 25,000 in the United States. While rare, it is currently the leading genetic cause of infant mortality. But,…

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Rising Cost of Drug Development isn’t Slowing Down Researchers Working to Uncover Novel Therapies for Rare Diseases

Every day the scientific community makes progress in the study of rare disease. Research in the field has advanced exponentially in recent years and we're starting to see more treatments…

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Nonprofit Organization Predicts Gene Therapy to be Most Cost Effective Treatment for Spinal Muscular Atrophy
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Nonprofit Organization Predicts Gene Therapy to be Most Cost Effective Treatment for Spinal Muscular Atrophy

According to a story from physiciansweekly.com, a recent report from the Institute for Clinical and Economic Review (ICER) suggests that, pending more data about overall price and longterm success rates,…

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Positive Results From a Phase IIb Trial of Firdapse for MuSK Myasthenia Gravis Published Online

Myasthenia Gravis (MG) is a progressive autoimmune disorder which results in the weakening of muscles. Most people with the condition are able to manage their symptoms with treatment and have…

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