Giuliana Grossi

Rhythm Pharmaceuticals shares data from long-term extension study of setmelanotide in Bardet-Biedl Syndrome.

ACR partners with the Vasculitis Foundation to establish a new guideline that addresses diagnostic issues relating to Kawasaki disease, a rare vasculitis that inflames the blood vessels.

Novartis announces promising results from research evaluating the safety and efficacy of Zolgensma.

More studies need to be done examining the associations of increased weight and outcomes among patients with cystic fibrosis.

Investigators examined the effect that pulsed low-intensity ultrasonography had on symptom relief and tibiofemoral articular cartilage thickness among patients with knee osteoarthritis.

Mindfulness-oriented recovery enhancement is unlike other mindfulness-based treatments and was found superior to supportive group psychotherapy for treating opioid misuse and chronic pain.

Policy expert, Cindy Steinberg, of the US Pain Foundation explains the updated changes that have been proposed by the CDC to the Clinical Guideline for Prescribing Opioids.

The clinical trial of efanesoctocog alfa provided positive top-line results for treating patients with severe Hemophilia

Transgender and nonbinary youths, a population with an exceptional risk of suicide and poor mental health outcomes, were found to have 60% lower odds of depression after receiving gender-affirming intervention.

The risk of death during, or shortly after pregnancy, was almost 3 times higher for Black women than for White women. Risk increased with age resulting in Black women 40 or over making up the majority of maternal deaths in 2020.

Sarcoidosis expert shares that one of her early patients had the rare disease and it was this young African American woman who inspired her to focus on treating the condition.

Investigators use an indirect approach to estimate the mortality rate of children with sickle cell anemia in sub-Saharan Africa because there's no systematic screening at birth.

Experts from the Violence Intervention Advocacy Program of the Boston Medical Center share insight from their research examining the spike of violence observed early in the COVID-19 pandemic.

In an interview, lead investigator and expert Dr. Martin Kolb discusses his new phase 2b HZN-825 trial and what it's like treating the rare disease.

The American College of Rheumatology releases 2 new guidelines for treating patients with juvenile idiopathic arthritis.

When the pandemic began, prescribers in Canada were allowed to increase the amount of opioid agonist therapy take-home doses for individuals with opioid use disorder.

New recommendations were released by ACR and AAHKS regarding perioperative management of antirheumatic medications for patients with rheumatic diseases undergoing these procedures.

Clinical results show that 98% of patients with relapsed or refractory multiple myeloma responded to cilta-cel after a single treatment.

Expert Mikkael Sekeres, MD, explains that the COVID-19 pandemic affected all of medicine, but in a rare disease domain that impact was even greater.

Expert John Sergent, MD, goes into detail about myositis and how to differentiate it from other inflammatory diseases.

These prominent leaders in the field of autoimmune disease discuss AHN's Autoimmunity Institute joining forces with the Relapsing Polychondritis Foundation.

Chief Scientific Officer of the CMT Research Foundation talks about being cognizant of the blood-nerve barrier when treating patients and developing therapies.

The president of Make-A-Wish New Jersey speaks about the impact of granting a wish to a child living with a rare disease.

The Rare Disease Report Podcast returns! Our first episode features Karen Kozarsky, PhD, an expert on gene therapy and adrenomyelonueropathy.

Why is Rare Disease Day so important? Hear the answers from some of the most qualified professionals in rare disease fields.

In this study investigators analyze the effects that a lifestyle program has on sleep and physical activity in older adults.

While recent data indicate the potential to reduce opioid overdose mortality rates with naloxone distribution, authors call for increased support for those who witness an overdose.

Vibeke Strand, MD, shares that based on her data, secukinumab is an effective option for treating patients with psoriatic arthritis.

The Chair of the ACR COVID-19 Vaccine Clinical Guidance Task Force shares what was learned from the Omicron variant and how the recommendations for rheumatologists has been affected by it.

Rare disease pyruvate kinase deficiency received first approved disease-modifying therapy.