Clinical Trials

Information and insight on the latest clinical trials
Farber Disease Natural History Study

Farber Disease Natural History Study

John Mitchell, MD, pediatric endocrinologist at Montreal Children’s Hospital and McGill University explains the need for a natural history study involving Farber disease.

WHIM Syndrome: Etiology and Diagnosis

WHIM Syndrome: Etiology and Diagnosis

  Philip M. Murphy, MD, Chief, Molecular Signaling Section, Laboratory of Molecular Immunology; Chief, Laboratory of Molecular Immunology, National Institute of Allergy and Infection, discusses the etiology and diagnosis of WHIM syndrome, a rare immunodeficiency...

WHIM Syndrome Clinical Trials

WHIM Syndrome Clinical Trials

Sarah Cohen, MD, Medical Director Rare Diseases, X4 Pharmaceuticals, discusses WHIM syndrome and clinical trials.

New ERT for Pompe Disease Showing Promise

New ERT for Pompe Disease Showing Promise

David Kronn, MD, Associate Professor of Pediatrics at New York Medical College describes the latest results from the mini-COMET phase II study testing avalglucosidase alfa to treat Pompe disease.

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