Between baseline and week 52, spinal canal dimensions numerically improved with navepegritide compared to placebo across L1 to L5. Weekly administration of navepegritide led to improvements in overall ...

The study will assess the number and severity of dose-limiting toxicities and adverse events. A new study will evaluate the safety and efficacy of universal allogeneic anti-CD19/BCMA CAR T-cells in ...

In patients who had undergone limb lengthening surgery, the annual growth velocity increased from 2.2 ± 0.7 to 6.5 ± 0.7 centimeters per year. Vosoritide significantly increased annual growth velocity ...

Questions remain regarding the exact relationship between AAV and inflammatory bowel diseases. A young adult female patient with a history of ulcerative colitis was subsequently diagnosed with ...

The findings from this study highlight the importance of further investigation of nailfold videocapillaroscopy when evaluating of patients with AAV in different age groups. In children with ...

For the last 3 years, the PBC Foundation, based in Edinburgh, Scotland, has conducted an annual summit that addresses the challenges and potential remedies for those living with primary biliary ...

The primary outcome measures include assessing I-RODS scores at baseline and monitoring variations at 6 and 12 months after enrollment An upcoming observational, noninterventional study aims to assess ...

A few weeks before I was diagnosed with chronic inflammatory demyelinating polyneuropathy (CIDP), I was very vulnerable. I lost many friends and was yanked out of the life I had expected to live. I ...

Patients with a history of myeloproliferative syndrome should be highly alert to the possibility of BCS in the presence of abdominal pain, ascites, and hepatomegaly. Polycythemia vera (PV) can ...

The levels of pNF-H and thigh skeletal muscle MRI can serve as potential biomarkers for evaluating the effectiveness of SMA treatment. The combination of rehabilitation therapy with nusinersen for the ...

The Muldoon family from Philadelphia, Pennsylvania, is shown after baby KJ’s infusion of a personalized gene-editing therapy (Photo courtesy of Children’s Hospital of Philadelphia) KJ Muldoon has been ...

The authors remarked that the results called for further education on MG symptoms for physicians outside the neurology setting. Advanced age and increased time between symptom onset appear to ...