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Sickle cell disease: symptoms, complications, diagnosis, treatment, and new advances in gene therapy

Sickle cell disease: symptoms, complications, diagnosis, treatment, and new advances in gene therapy

by Dr. Gabriel M. Freitas | 11/04/2025 | Genetic and Hereditary Diseases

Content discussed in this post What sickle cell disease is Symptoms and how the disease presents across the lifespan Complications that require immediate attention Sickle cell trait vs sickle cell disease Diagnosis, newborn screening, and tests Day-to-day treatment...
Spinal Muscular Atrophy (SMA): symptoms, types, diagnosis, treatment, and quality of life

Spinal Muscular Atrophy (SMA): symptoms, types, diagnosis, treatment, and quality of life

by Dr. Gabriel M. Freitas | 10/09/2025 | Genetic and Hereditary Diseases, Neurological Diseases

Content discussed in this post What SMA is and why it matters Symptoms and signs by age group SMA types and the role of SMN2 Diagnosis and newborn screening Disease-modifying treatment Respiratory and nutritional support Physical therapy, speech-language pathology,...
Huntington’s Disease: symptoms, genetic causes, diagnosis, treatment, and what’s new

Huntington’s Disease: symptoms, genetic causes, diagnosis, treatment, and what’s new

by Dr. Gabriel M. Freitas | 09/30/2025 | Genetic and Hereditary Diseases, Neurological Diseases

Content covered in this post What Huntington’s disease is Symptoms: motor, cognitive, and behavioral Causes and heredity (HTT/CAG) Diagnosis and key differentials Treatment and multidisciplinary care Lifestyle and safety Research and advances Quick FAQ Health...
Duchenne Muscular Dystrophy: Causes, Symptoms, Diagnosis, and Treatment

Duchenne Muscular Dystrophy: Causes, Symptoms, Diagnosis, and Treatment

by Dr. Gabriel M. Freitas | 09/09/2025 | Genetic and Hereditary Diseases

Duchenne Muscular Dystrophy (DMD) is a rare and progressive genetic disorder that primarily affects boys. It is characterized by progressive muscle weakness caused by the absence or insufficient production of a protein called dystrophin, which is essential for the...
Gaucher Disease: Symptoms, Causes, and Treatment

Gaucher Disease: Symptoms, Causes, and Treatment

by Dr. Gabriel M. Freitas | 08/16/2025 | Genetic and Hereditary Diseases

Gaucher disease is a rare genetic condition that primarily affects the liver, spleen, and bone marrow, but it can also impact several other organs in the body. Although not widely known, it is the most common lysosomal storage disorder, caused by a mutation in a...
Cystic Fibrosis: Causes, Symptoms, Treatment, Myths, and Quality of Life

Cystic Fibrosis: Causes, Symptoms, Treatment, Myths, and Quality of Life

by Dr. Gabriel M. Freitas | 07/16/2025 | Genetic and Hereditary Diseases, Respiratory Diseases

Cystic fibrosis (CF), also known as mucoviscidosis, is a rare and chronic genetic disease that primarily affects the lungs and digestive system. Although it poses significant challenges, advances in medicine have transformed the outlook for many individuals, enabling...
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