The First Patient has Been Dosed with an Investigative Therapy for Acute Myeloid Leukemia

Immune-One Therapeutics has just announced that they have dosed their first acute myeloid leukemia (AML) patient in their Phase 1 trial investigating IO-202. This therapy is an immune inhibitory receptor that works…

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New Reduced-Intensity Conditioning for HSCT Found Beneficial for 20 Rare Diseases

A recent study published in the journal Blood Advances has documented that hematopoietic stem cell transplantation (HSCT) is both safe and effective for children with many different kinds of inherited nonmalignant conditions…

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New Investigative Therapy for Hereditary Angioedema Found Efficacious in Study

Ionis Pharmaceuticals has just released their findings from a study investigating the effect of IONIS-PKK-LRx and IONIS-PKKRx in hereditary angioedema (HAE) patients with different forms of the disease. These therapies work to reduce…

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Study Finds New Therapy to be Efficacious for Rheumatoid Arthritis Patients Unresponsive to Methotrexate

A new study published in the journal Rheumatology and Therapy examined the effect of baricitinib on Rheumatoid Arthritis (RA) patients who have had an inadequate to their methotrexate therapy. Methotrexate and Baricitinib…

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Heterozygous Familial Hypercholesterolemia Trial Shows Positive Results of Therapy in Teenagers

Researchers in Canada have just released positive results from their heterozygous familial hypercholesterolemia (HeFH) clinical trial. This investigation was examining the effect of Repatha on teenagers with the condition. The trial…

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Noninvasive Genetic Testing for Keratoconus Helps Identify Who is at Risk
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Noninvasive Genetic Testing for Keratoconus Helps Identify Who is at Risk

A new genetic test for keratoconus as well as other types of corneal dystrophy was released late last year. The test was created by Avellino and can identify biomarkers of disease. It also looks…

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Progressive Familial Intrahepatic Cholestasis Online Support Groups Provide Comfort During COVID-19

Claire Brinkley Claire Brinkley's daughter Eleanor is diagnosed with progressive familial intrahepatic cholestasis (PFIC) which is a rare disease affecting the liver. As a newborn, she appeared perfectly normal. But at…

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Virtual Event September 10th for Myeloproliferative Neoplasm International Awareness Day

Myeloproliferative neoplasm (MPN) International Awareness Day is on September 10th. This year, there will be a live streaming event to help bring awareness to this condition, instead of an in-person…

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Molecular Discovery Will Lead to Greater Understanding of X-Linked Genetic Diseases Like Rett Syndrome
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Molecular Discovery Will Lead to Greater Understanding of X-Linked Genetic Diseases Like Rett Syndrome

Female mammals have XX chromosomes and males have XY. But scientists have never understood how females decide which of their two X's should be active in each of its cells.…

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Research Has Been Accelerated to Understand the Association of Chronic Fatigue Syndrome and COVID-19

Dr. Harvey Moldofsky Harvey Moldofsky is a professor emeritus at the University of Toronto. His area of focus is SARS, and since the onset of COVID-19, he has been fully…

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Chronic Lyme Disease Patient Provides Advice for People Experiencing Long-Lasting Effects of COVID-19
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Chronic Lyme Disease Patient Provides Advice for People Experiencing Long-Lasting Effects of COVID-19

Ross Douthat, a writer for the New York Times, was first diagnosed with Lyme disease and a Bartonella infection in 2015 from a deer tick bite. The effects have lingered…

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European Commission Grants Orphan Designation Designation for Investigative Achondroplasia Therapy
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European Commission Grants Orphan Designation Designation for Investigative Achondroplasia Therapy

Ascendis Pharma has just announced that their investigative therapy for achondroplasia called TransCon CNP has just received Orphan Drug Designation from the European Commission. This same designation has already been…

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ICYMI: Phase 3 Study for Dermatomyositis has Completed Enrollment
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ICYMI: Phase 3 Study for Dermatomyositis has Completed Enrollment

Corbus Pharmaceuticals has just announced that participant enrollment for their Phase 3 trial of lenabasum for dermatomyositis has been completed. Dermatomyositis Dermatomyositis (DM) is a rare autoimmune disease. It can…

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The Brighter Days Care Package Program for Cystinosis Patients and Families has Launched
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The Brighter Days Care Package Program for Cystinosis Patients and Families has Launched

The Cystinosis Research Network (CRN) has created a new program to bring light to cystinosis families during these uncertain times. It is called the Brighter Days Care Package Program. Brighter…

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How COVID-19, Chronic Fatigue Syndrome, and Guillain-Barre Syndrome are Linked
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How COVID-19, Chronic Fatigue Syndrome, and Guillain-Barre Syndrome are Linked

We have learned a lot, but still have much to learn about COVID-19. Researchers have seen a link between COVID-19 and chronic fatigue syndrome (myalgic encephalomyelitis) as well as Guillain-Barre syndrome, which…

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Correlation Between Marfan Syndrome and Pregnancy Related Arterial Dissections
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Correlation Between Marfan Syndrome and Pregnancy Related Arterial Dissections

Arterial Dissection Arterial dissections refer to the tearing of the inner wall of an artery. Pregnancy creates stress on the body's blood vessels and in rare cases dissections can occur.…

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ICYMI: Better Research Means Better Outcomes for Charcot-Marie-Tooth Disease Patients
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ICYMI: Better Research Means Better Outcomes for Charcot-Marie-Tooth Disease Patients

Researchers have been trying for years to better understand the mutations which cause the rare disorder called Charcot-Marie-Tooth disease (CMT). Tremendous progress has been made. But now that we know…

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