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Vertex Announces FDA Approvals of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor), Symdeko (tezacaftor/ivacaftor and ivacaftor) and Kalydeco (ivacaftor) for Use in People With CF With Certain Rare Mutations
2021-01-17 Mark Gualtieri Health News

BOSTON–(BUSINESS WIRE)–December 21, 2020 — Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) expanded the eligibility for Trikafta (elexacaftor/tezacaftor/ivacaftor

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Hope for children with rare heart condition: Novel stem cell therapy to save the day
2021-01-12 Mark Gualtieri Health News

Dilated cardiomyopathy (DCM) is a condition caused by the weakening of the heart muscle, affecting the ventricles (chambers in the heart that push blood around

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New drug molecules hold promise for treating rare inherited terminal childhood disease
2020-12-18 Mark Gualtieri Health News

Scientists at the University of Exeter have identified a way to “rescue” cells that have genetically mutated, paving the way to a possible new treatment

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See Madonna's Rare Look at All 6 Kids in Family Video: 'Giving Thanks'
2020-12-01 Mark Gualtieri Health News

All here! Madonna gave a rare glimpse of her six children in a Monday, November 30, Instagram video. Full House! Jude Law and More Celeb

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Understanding rare type of diabetes gives insights into mechanisms of insulin production
2020-11-16 Mark Gualtieri Health News

Solving the genetic puzzle of why babies developed a rare type of diabetes has uncovered a new biological pathway that is fundamental to insulin production,

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Providing new hope for children suffering from rare muscle diseases
2020-10-09 Mark Gualtieri Health News

Stephen Greenspan and Laura Zah were devastated when they learned their son Alexander had a rare genetic mutation, which causes a deadly neuromuscular disease with

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Coronavirus test caused brain fluid leak in woman with rare health condition: report
2020-10-02 Mark Gualtieri Health News

American citizens arrested for not wearing masks outdoors: Author Alex Berenson, author of ‘Unreported Truths About COVID-19 and Lockdowns Part 2’ weighs in on the

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In Rare Case, COVID-19 Test Caused Spinal Fluid Leak
2020-10-01 Mark Gualtieri Health News

THURSDAY, Oct. 1, 2020 — Doctors stress that it’s a very rare occurrence, but one woman’s pre-surgery COVID-19 nasal swab test appears to have triggered

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13-year-old dies of rare ‘brain-eating’ amoeba after swimming in Florida lake
2020-09-13 Mark Gualtieri Health News

A teen has died from a rare “brain-eating” amoeba infection after a family vacation in Florida, according to news reports. The 13-year-old, Tanner Wall, and

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Reducing urinary protein for patients with rare kidney disease slows kidney decline
2020-08-10 Mark Gualtieri Health News

Reducing the amount of protein in the urine of patients with focal segmental glomerulosclerosis (FSGS), a rare disease in which scar tissue forms on the

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