Articles related to RARE CONDITIONS & DISEASES

Less than half of the 26,000 annual rhabdomyolysis cases in the United States are exercise-related, but early recognition remains crucial for preventing severe complications.

The study reveals that 10% of patients treated with eli-cel developed hematologic cancer, with an incidence rate of 2.1 per 100 person-years. This finding emphasizes the need for careful monitoring and risk assessment in gene therapy approaches.

AL amyloidosis can present with distinctive periorbital signs, including petechiae and waxy eyelid papules, which may worsen with trauma.

Yale researchers have developed an AI model capable of diagnosing Marfan syndrome from facial photographs with 98.5% accuracy, potentially enabling earlier intervention for this life-threatening genetic disorder.

The Social Security Administration’s expansion of the Compassionate Allowances program now covers 287 severe health conditions, streamlining the disability benefits process for patients with urgent medical needs.

In a phase 3 trial, oral sebetralstat demonstrated a median time to beginning of symptom relief of 1.61 hours for the 300 mg dose and 1.79 hours for the 600 mg dose, compared to 6.72 hours for placebo in hereditary angioedema attacks.