Giuliana Grossi

Bristol Myers Squibb has to wait 3 months for FDA decision on Reblozyl for treating non-transfusion-dependent β-thalassemia.

Interstitial lung disease expert explains the importance of educating patients with autoimmune disease.

Patients with Fabry disease who received treatment with PRX-102 every 4 weeks displayed a stable condition at the end of the trial.

A summarization of the in-depth review, editorial and synopsis provided by multiple experts after revising the updates to the VA/DoD joint clinical practice guideline for substance use disorders.

In 2020 many clinics shifted to telehealth or reduced-contact practices which applied to medication abortions. Physicians relied solely on history-based screening instead of ultrasonography or pelvic exams during this time and investigators analyzed the data.

The use and legalization of cannabis products has been increasing in the US for a variety of medical concerns. Investigators aim to better understand the risks and benefits of owning a medical marijuana card.

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

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.

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.

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.

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.

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.

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.

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

The United Nations 2030 Agenda for Sustainable Development has called for an end to violence against women in its Sustainable Development Goals.

Intravenous tramadol, developed by Avenue Therapeutics, fails to attain FDA approval as treatment for acute pain in adults.

Progressive familial intrahepatic cholestasis is a genetic liver disease that doesn't only affect the children who are diagnosed, but also the caregivers responsible for them.