tag rare-disease-day 
STAT News
OCTOBER 6, 2022
Rise and shine, everyone, another busy day is on the way. Have a wonderful day, and do keep in touch. million tag as cost effective for a Bluebird Bio drug called Zynteglo for the blood disorder beta thalassemia, Arbuckle said in an interview. Now, though, we have returned and there is much to do. So time to get cracking.
XTalks
DECEMBER 13, 2023
The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. Casgevy is also the first ever CRISPR/Cas9-based therapy approved in the US.
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pharmaphorum
NOVEMBER 19, 2021
Roche’s oral treatment Evrysdi for the rare genetic disease spinal muscular atrophy (SMA) will be made available on the NHS in England, after NICE reached a three-year access agreement with the company. The drug, which is administered as an oral liquid once a day after meals, costs almost $8,000 per bottle.
pharmaphorum
OCTOBER 21, 2021
“Every day that passes we estimate that over a thousand Americans move from mild to moderate Alzheimer’s dementia, and therefore may no longer be appropriate for initiation of treatment with Aduhelm,” he said on a conference call.
XTalks
FEBRUARY 27, 2024
Rare Disease Day 2024, which falls on February 29 this year, is an opportunity to unite under a common cause: to bring attention to the challenges faced by those living with rare diseases and to push for advancements in research, treatment and policy.
The Pharma Data
OCTOBER 21, 2020
21, 2020 — A combination of two “targeted” therapies can beat back a rare form of blood cancer — without the toxic effects of chemotherapy, a new study has found. The disease is a subtype of ALL in which the cancer cells have a genetic abnormality called the Philadelphia chromosome (Ph). WEDNESDAY, Oct.
The Pharma Data
OCTOBER 21, 2020
21, 2020 — A combination of two “targeted” therapies can beat back a rare form of blood cancer — without the toxic effects of chemotherapy, a new study has found. The disease is a subtype of ALL in which the cancer cells have a genetic abnormality called the Philadelphia chromosome (Ph). WEDNESDAY, Oct.
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