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Mashup Score: 2
The NFL says it will allow players to wear protective soft-shell helmet covers known as…
Source: www.houstonchronicle.comCategories: General Medicine News, Rare DiseaseTweet
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Mashup Score: 0
Ten years after dipping its toe into the gene therapy pool in a licensing deal with Spark Therapeutics, Pfizer has gained an FDA approval for the acquired treatment. | Ten years after dipping its toe into the gene therapy pool in a licensing deal with Spark Therapeutics, Pfizer has gained an FDA approval for the acquired treatment. The U.S. regulator has endorsed Beqvez for adults with the bleeding disorder hemophilia B. It becomes the first FDA-approved gene therapy for Pfizer, which will charge $3.5 million for the single-dose drug.
Source: www.fiercepharma.comCategories: General Medicine News, Rare DiseaseTweet
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Mashup Score: 0ETSU ALS clinic wins $75K grant to expand its free care services - 10 hour(s) ago
The clinic at East Tennessee State University Health is the only one in the state to offer free multidisciplinary care to ALS patients.
Source: alsnewstoday.comCategories: General Medicine News, Rare DiseaseTweet
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Mashup Score: 3Scientists report that new gene therapy slows down amyotrophic lateral sclerosis disease progression - 10 hour(s) ago
There has been a breakthrough in the research on the disease amyotrophic lateral sclerosis (ALS). Scientists at Umeå University report that the disease progression in a patient with a particularly aggressive …
Source: medicalxpress.comCategories: General Medicine News, Rare DiseaseTweet
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Mashup Score: 0For Beaten-Down Maker of Alzheimer’s Drug, Good Enough Will Do - 10 hour(s) ago
In-line sales for Alzheimer’s drug Leqembi and a beat for rare-disease drug Skyclarys has Wall Street giving Biogen another chance.
Source: www.wsj.comCategories: General Medicine News, Rare DiseaseTweet
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Mashup Score: 5Events | EverythingALS - 11 hour(s) ago
EverythingALS is a patient-focused non-profit, part of Peter Cohen Foundation (PCF) a 501(3)c organization. Our mission is to support efforts to care for ALS patients and work to find a cure by creating a platform for direct engagement with patients, caregivers, advocates, and researchers.
Source: www.everythingals.orgCategories: General Medicine News, Rare DiseaseTweet
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Mashup Score: 8
SACRAMENTO, CA, April 24, 2024—Today, California is one step closer to establishing a Rare Disease Advisory Council (RDAC) to help the 1-in-10 Californians
Source: rarediseases.orgCategories: General Medicine News, Rare DiseaseTweet-
#California is one step closer to establishing a #RareDisease Advisory Council! Earlier this week, the bill to establish an #RDAC, introduced by @AsmRickZbur, passed out of committee, a major milestone. Learn more and take action to move the bill forward: https://t.co/N8CXSUmZCA https://t.co/LLxfwKRd7Y
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Mashup Score: 6Rare Impact Awards - NORD's Rare Impact Awards - 1 day(s) ago
This very special event will celebrate NORD’s legacy and achievements, and honor those individuals and organizations doing remarkable work on behalf of the rare disease community.
Source: rareimpact.orgCategories: General Medicine News, Rare DiseaseTweet
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Mashup Score: 39Thrombocytosis - 1 day(s) ago
An asymptomatic 55-year-old woman presented with thrombocytosis discovered 2 years earlier. Laboratory testing revealed the following: hemoglobin 13.1 g/dL, red blood cell count 5.04 × 10 9 /L, hematocrit 41.8%, mean corpuscular volume 83 fL, mean corpuscular hemoglobin concentration 31.3 g/dL, leukocytes 10.87 × 10 9 /L (neutrophils 43%, lymphocytes 45%, monocytes 5%, eosinophils 4%), and platelets 513 × 10 9 /L. Serum ferritin levels were low at 8 ng/mL. Physical examination was unremarkable. Serum
Source: ashpublications.orgCategories: General Medicine News, Rare DiseaseTweet
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Mashup Score: 0Enhanced Health Equity and Timely Diagnoses in Rare Diseases - 2 day(s) ago
Cheryl Schwartz discusses Takeda’s report that calls for enhanced health equity and timely diagnosis for rare disease patients
Source: CheckRare.comCategories: General Medicine News, Rare DiseaseTweet
RT @BrielmaierL: NFL to allow players to wear protective soft-shell helmet covers during games https://t.co/PukdDxxDte via @HoustonChron…