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Sangamo and Pfizer partner for development of new zinc finger protein gene therapy to treat ALS

Sangamo and Pfizer partner for development of new zinc finger protein gene therapy to treat ALS

New, sensitive screening test may allow clinicians to accurately detect Pompe disease

New, sensitive screening test may allow clinicians to accurately detect Pompe disease

FDA chief says he’s open to rethinking incentives on orphan drugs

FDA chief says he’s open to rethinking incentives on orphan drugs

Study: People with rare diseases at high risk for experiencing poor quality of life

Study: People with rare diseases at high risk for experiencing poor quality of life

Moderate exercise reduces progression of Marfan syndrome symptoms in mice

Moderate exercise reduces progression of Marfan syndrome symptoms in mice

A guide to Severe Combined Immunodeficiency Disease, review released

A guide to Severe Combined Immunodeficiency Disease, review released

New review provides guidance to doctors who may treat infants with ‘bubble boy disease’

New review provides guidance to doctors who may treat infants with ‘bubble boy disease’

Experts tell Congress how to cut drug prices

Experts tell Congress how to cut drug prices

Patients with rare diseases and congress square off over orphan drug tax credits

Patients with rare diseases and congress square off over orphan drug tax credits

Study finds lack of follow-up care for more than two-thirds of children with concussion

Study finds lack of follow-up care for more than two-thirds of children with concussion

Researchers discover mechanism behind skin inflammation in eczema

Researchers discover mechanism behind skin inflammation in eczema

New gene therapy trial for X-linked myotubular myopathy offers hope to children

New gene therapy trial for X-linked myotubular myopathy offers hope to children

FDA approves new drug Zelboraf for Erdheim-Chester disease

FDA approves new drug Zelboraf for Erdheim-Chester disease

House Republicans aim to yank tax credits for orphan drugs

House Republicans aim to yank tax credits for orphan drugs

HPV vaccination and screening combination can decrease cancer risk by more than 90%

HPV vaccination and screening combination can decrease cancer risk by more than 90%

Biogen and Ionis awarded 2017 Prix Galien USA Award for SPINRAZA

Biogen and Ionis awarded 2017 Prix Galien USA Award for SPINRAZA

Key features that distinguish Brittle Type 1 Diabetes from stable T1D

Key features that distinguish Brittle Type 1 Diabetes from stable T1D

WHO helps contain Marburg virus disease outbreak on Uganda-Kenya border

WHO helps contain Marburg virus disease outbreak on Uganda-Kenya border

Study to determine lowest dose of chemotherapy needed for babies with SCID

Study to determine lowest dose of chemotherapy needed for babies with SCID

First-ever treatment for Merkel cell carcinoma receives approval in Japan

First-ever treatment for Merkel cell carcinoma receives approval in Japan