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Spinal muscular atrophy

Woman sleeping withy tablet on night table
Informatics

Broadening the Scope of Clinical Trials: The Changing Role of Real-World Evidence

Helen Albert -
0
Futuristic flight through a digital line landscape, Blue dust particle
Informatics

Real-World Data: The Secret Sauce for Rare Disease Research?

Malorye Branca -
0
PacBio’s Sequel
Molecular Dx

PacBio, Twist Launch Off-the-Shelf Long-Read Panel Line

Chris Anderson -
0
Jonathan Thon with colleagues
News & Features

Asked and Answered: Jonathan Thon Talks about Bringing Gene Therapy to Life

Damian Doherty -
0
Little girl receives breathing treatment
Molecular Dx

Antisense Potential Treatment for Cystic Fibrosis

Malorye Branca -
0
A genotype-to-phenotype approach to discovering a new autoinflammatory disease
Molecular Dx

In-Depth Sequencing Project Benchmarks Medically Relevant Genetic Variants

Helen Albert -
0
Rady Children’s and Takeda Partner to Develop Treatments for Rare Genetic Diseases
Molecular Dx

Rady Children’s and Takeda Partner to Develop Treatments for Rare Genetic Diseases

Helen Albert -
0
Neurons sending pulsating electric impulses and communicating with each other. Concept For Neurological Diseases, Tumors And Brain Surgery
News & Features

Next Steps for Huntington’s Therapeutics Field After Roche, Wave Failures

Helen Albert -
0
Nerve cells of the human brain
Molecular Dx

RNA Metabolism Gene Mutation Linked to Rare Disease

Malorye Branca -
0
DNA Sequencing Data
Molecular Dx

Human Genome Project at 20

Anjali A. Sarkar, PhD -
0
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