Articles

Podcasts

Videos

The approval is based on 24- and 80-week findings showing significant benefit in LDL-C reduction with the subcutaneous injection drug.

FDA Approves Evolocumab for Pediatric Familial Hypercholesterolemia Indications

New study finds that telehealth is a viable option for treating sickle cell anemia patients in rural populations. 

Evaluating the Impact of Telehealth on Hydroxyurea Response

Voxelotor has the potential to decrease long-term problems in pediatric sickle cell disease. 

Kim Smith-Whitley, MD: The Impact of Voxelotor on Pediatric Sickle Cell

Kim Smith-Whitley, MD, discussed her research on voxelotor as treatment for children with sickle cell disease.

Kim Smith-Whitley, MD:  Voxelotor and Sickle Cell Disease

Voxelotor study results show it’s just as effective in children as adults and adolescents. 

Kim Smith-Whitley, MD:  New Treatment for Children with Sickle Cell Disease

Obstructive sleep apneas and overnight hypoxemia are more likely to develop in patients with sickle cell disease.

Pediatric Sickle Cell Disease Linked to Sleep Disorder Complications

The Firmicutes/Bacteroidetes ratio was lower for patients with sickle cell disease compared to a healthy control group. 

Gut Diversity Could Yield Better Treatments for Sickle Cell Disease

Ketamine was not superior to morphine in reducing pain scores.

Ketamine Results in Drop in Pain Scores for Sickle Cell Patients

The main mechanism for exercise intolerance is impaired blood oxygen carrying capacity.

Investigators Seek Explanations for Exercise Intolerance for Sickle Cell Patients

Niemann-Pick is highly heterogenous and is characterized by hypervariable manifestations across patients. 

Sharon Hrynkow, PhD: A Look at Niemann-Pick Disease

Sickle Cell

Sickle Cell condition center page, resources on the topics of medical news and expert insight into sickle cell disease can be found. Content includes articles, interviews, videos, podcasts, and breaking news on sickle cell research, treatment, and drug development.

Amyloidosis

The HCPLive amyloidosis page is a resource for medical news and expert insights on the rare disease. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for amyloid buildup, and more.

Duchenne Muscular Dystrophy

On the HCPLive Duchenne muscular dystrophy page, resources on the topics of medical news and expert insight into DMD can be found. Content includes articles, interviews, videos, podcasts, and breaking news on the rare disease, and more.

Huntington Disease

On the HCPLive Huntington disease page, resources on the topics of medical news and expert insight into HD can be found. Content includes articles, interviews, videos, podcasts, and breaking news on Huntington's chorea, and more.

Lysosomal Storage Disorders

Lysosomal storage disorders page, resources on the topics of medical news and expert insight into the rare diseases can be found. Content includes articles, interviews, videos, podcasts, and breaking news on lysosomal disorder disease research, treatment, and drug development.

The Rare Disease Report page is a resource for medical news and expert insights on rare diseases. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for sickle cell disease, multiple myeloma, and more.

RDR Alert®