Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disorder that leads to progressive weakness and loss of movement. SMA results from the degeneration of alpha motor neurons in ...

Follow Rare Disease Advisor's Hemolytic Disease of the Fetus and Newborn (HDFN) Junction for the latest HDFN news, insights, and resources.

Get the latest news, resources, and perspectives on Duchenne muscular dystrophy (DMD), written specifically for HCPs who treat the disease.

Are you a healthcare provider looking for information on sickle cell disease histology? Get a brief, comprehensive overview here.

Recurrent hypoglycemic coma episodes may lead to a primary biliary cholangitis diagnosis.

Are you a healthcare provider searching for information on spinal muscular atrophy type 4? Read about SMA type 4 here.

From landmark gene therapy approvals to illuminating discoveries into the pathogenesis of rare diseases, 2023 saw substantive advances in rare disease diagnosis and treatment. The following list of ...

Looking for information about Cold Agglutinin Disease? Get the latest news, resources & insights into treatment of CAD here.

I was diagnosed with generalized myasthenia gravis (MG) in August 2019. Prior to my diagnosis, I was the head of global sales and business development for a life science consulting firm. For nearly 10 ...

Are you a health care provider looking for information on sickle cell disease life expectancy? Read more about it here.

Vyndamax® (tafamidis) is an oral therapy developed by Pfizer that is indicated for the treatment of cardiomyopathy in adults with wild-type or hereditary ...

Follow Rare Disease Advisor's Sickle Cell Disease Junction for the latest news, resources, insights, and patient perspectives on the disease.