Overcoming the Clinical Challenge of Detecting and Diagnosing LAL-D
Lysosomal acid lipase deficiency is so rare that most doctors have never heard of it, let alone treated a patient with the disease, says Henry Lin, MD, MBA.
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Rare Disease Day 2022: Health Care Policy and Protections Still Lacking in Many States
NORD and other rare disease organizations around the world will celebrate Rare Disease Day on February 28, 2022, with most events being held virtually.
Using Technology to Boost Ethnic, Geographic Diversity in Clinical Trials
Eliminating geographic and ethnic bias in clinical trials for rare diseases is the goal of Jeeva Informatics, says founder and CEO Harsha K. Rajasimha, PhD.
CORD Aims to Improve Diagnosis, Drug Access for Canada’s Rare Disease Patients
The Canadian Organization for Rare Disorders advocates for better diagnosis and drug access for Canada’s 3 million people with rare diseases.
Gene Therapy May Offer Hope for Patients With LAL-D, a Disease With No Known Cure
Researchers are investigating gene therapy for use in the treatment of lysosomal acid lipase deficiency (LAL-D).
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Israel Emerges as Global Powerhouse in Rare Disease Research
When it comes to rare disorders, Israel—a New Jersey-sized nation of 9.3 million that’s made headlines this year for its highly successful coronavirus vaccination campaign—is clearly an emerging powerhouse.
LAL-D Therapy Proves to Be a Lifesaver for 4 Michigan Brothers With the Rare Disease
Lysosomal acid lipase deficiency (LAL-D) affects fewer than 1 in 50,000 people. But when it comes to rare diseases, Michigan schoolteacher Rebecca Trendy seems to have hit the jackpot. All 4 of her sons have LAL-D — an inherited liver disease that renders the body unable to digest fats. “It’s been quite the medical journey…
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NORD Recognizes Progress in Fight Against Rare Disease With 2021 Rare Impact Awards
Under COVID-19’s lingering shadow, the National Organization of Rare Disorders (NORD) has presented its 2021 Rare Impact Awards to 24 pharmaceutical firms, researchers, politicians, and nonprofit groups for their efforts over the past year on behalf of patients with such illnesses.
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FDA, CDC Urge COVID-19 Vaccines for Rare Disease Patients on NORD Webinar
As the super-contagious Delta variant of COVID-19 sweeps across the globe, driving up infection rates as well as deaths, top US health officials are urging everyone 12 and older—including the country’s 30 million or so rare disease patients—to get vaccinated quickly.
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