Rare Diseases

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Rare Diseases

What Is Cystadane? Cystadane (betaine) is a prescription medication used to treat homocystinuria—a rare genetic disorder that causes a buildup of homocysteine in your body. Too much homocysteine is toxic and can lead to numerous

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Givlaari (Givosiran) – Subcutaneous

What Is Givlaari? Givlaari (givosiran) is an injectable prescription medication used to treat adults with acute hepatic porphyria (AHP). AHP is a group of rare genetic conditions in which there's not enough of a special liver enzyme that

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What Is Distal Muscular Dystrophy?

Distal muscular dystrophy is the name of a group of progressive disorders that leads to weakness in the distal muscles. Distal muscular dystrophy (DD), also called distal myopathy, is not one disorder, but a group of genetic disorders under the larger umbrella of muscular dystrophy. DD is marked by progressive weakness and/or wasting of the voluntary distal

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Syringomyelia: Overview and More

Syringomyelia is a disorder in which a fluid-filled cyst forms within the spinal cord. Over time, the cyst can enlarge and may damage the spinal cord. Syringomyelia is a rare disease in which a fluid-filled cyst, or syrinx, forms in the spinal cord. Over time, the syrinx can grow and compress on the spinal cord, which can interfere with the body's ability to send information to and from the

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What Is Aplasia?

Aplasia is the congenital failure of a limb, organ or other body part to develop. Learn about different types of aplasia, their symptoms, and causes. Aplasia is a medical term meaning that some part of the body is absent after it should already have developed. It comes from the roots “a,” (meaning without) and the Greek “plasia” (meaning formation).Aplasia isn’t a single medical

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Emflaza (Deflazacort) - Oral

What Is Emflaza? Emflaza (deflazacort) is a prescription drug used to treat a rare genetic muscle-wasting disease called Duchenne muscular dystrophy (DMD). It is part of a drug class known as corticosteroids and can be used in people aged 2

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What Is Gorlin Syndrome?

Gorlin syndrome is a rare genetic disease that causes symptoms such as basal cell carcinomas and tumors. It can be challenging, but isn't fatal Gorlin syndrome is a rare genetic disorder that often runs in families. This condition is caused by a mutation in a gene that suppresses the formation of tumors. Gorlin syndrome can affect any organ in the body, including the skin, bones, and

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What Is Gene Therapy?

Learn about gene therapy, which attempts to cure disease or improve the body's ability to fight it by adding a new gene or replacing a faulty gene. Gene therapy is a type of treatment being developed to fight diseases that are caused by genetic defects. This is a relatively new medical intervention that is mainly in the experimental phase, including human trials and animal trials, for the

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Causes and Risk Factors of Fanconi Anemia

The exact cause of Fanconi anemia (FA) is unknown, but research has uncovered at least 16 genes that are linked to the disease. Learn more. Fanconi anemia (FA) is a rare hereditary disease. About 50% of cases are diagnosed before the age of 10, and an estimated 10% are diagnosed in adulthood.The exact cause of FA is unknown, but research has shown that there is a strong genetic

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What Neuroscientists & Dietitians Swear By For All-Day Focus & Attention

Benefits include sustained mental and physical energy, concentration, and productivity.*

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CDC Recommends COVID Boosters for 5- to 11-Year-Olds

Kids ages 5 to 11 can now get a booster shot of the COVID vaccine. Learn how the organization made this decision, and what it means for families. Key TakeawaysThe Centers for Disease Control and Prevention now recommends Pfizer booster shots for children ages 5 and older.Pediatricians say boosters are important to keep young children safe from the virus, but more children need to get their

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