Rose McNulty

Activities of daily life, education, and employment were areas of difficulty in the transition to adulthood for patients with Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD), but those who had more siblings reported being more ready to transition […]

Ten children with profound genetic hearing loss due to variants of the otoferlin (OTOF) gene experienced clinically meaningful hearing improvements after receiving DB-OTO gene therapy, according to results presented at the Association for Research in Otolaryngology’s (ARO) 48th Annual ARO MidWinter

Patients with compensated metabolic dysfunction–associated steatohepatitis (MASH) cirrhosis who received resmetirom (Rezdiffra; Madrigal Pharmaceuticals) experienced reductions in liver stiffness in the phase 3 MAESTRO-NAFLD-1 trial (NCT04197479), according to 2-year results announced in a press release.1 In the MAESTRO-NAFLD-1 trial, liver

Patients with Duchenne muscular dystrophy (DMD) treated with SGT-003, an investigational gene therapy, showed microdystrophin expression at 90 days post treatment in the INSPIRE DUCHENNE trial (NCT06138639), Solid Biosciences announced in a press release.1 The initial data from the phase

Using anti-acetylcholine receptor antibody (AChR-Ab) titers to predict myasthenia gravis severity and improvement is controversial, but there was a correlation between decreased AChR-Ab titer and myasthenia gravis symptom scores in a recent study published in Frontiers in Neurology.1 AChR-Ab titers

The next-generation sequencing–based AlloHeme test demonstrated accuracy and sensitivity in predicting relapse following allogeneic hematopoietic stem cell transplantation (allo-HSCT) in acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS), according to new data from the prospective ACROBAT study (NCT04635384) presented at

The FDA today approved Amneal Pharmaceuticals’ generic lenalidomide capsules in multiple strengths, the company announced. Amneal will be granted a license to manufacture and sell the product, which references Celgene’s Revlimid, beginning in 2026. Amneal Pharmaceuticals’ generic lenalidomide capsules were

Patient-reported outcomes (PROs) were similar among those treated with tivozanib monotherapy and those given combination tivozanib and nivolumab for renal cell carcinoma (RCC) in the TiNivo-2 study (NCT04987203), according to a poster presented at the American Society of Clinical Oncology

Nerandomilast met its primary end point of absolute change from baseline in forced vital capacity (FVC) vs placebo in the FIBRONEER-ILD trial (NCT05321082), which assessed the agent in patients with progressive pulmonary fibrosis (PPF).1 Boehringer Ingelheim will submit a new

While cell and gene therapies (CGTs) can be life-changing for patients with complex and rare conditions, patients and caregivers face challenges in the pursuit of these treatments, according to a white paper from the National Pharmaceutical Council (NPC).1 The paper,