Global pharma company with U.S. HQ in Atlanta to make nearly $2B acquisition
UCB has agreed to buy Zogenix, a California-based biopharmaceutical company that develops treatments for rare diseases. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - January 19, 2022 Category: Biotechnology Authors: Maegan Smith Source Type: news

Beam Therapeutics inks deal with Pfizer worth up to $1.35B
Gene-editing firm Beam Therapeutics Inc. has signed a four-year deal worth up to $1.35 billion with pharmaceutical giant Pfizer Inc. Pfizer (NYSE: PFE) is paying Cambridge-based Beam (Nasdaq: BEAM) $300 million up front to develop gene therapies for three targets for rare diseases in the liver, muscle and central nervous system. Milestone payments to Beam could reach up to $1.05 billion. Pfizer will retain the options on exclu sive, global licenses for each development candidate; if it exercises… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - January 10, 2022 Category: Biotechnology Authors: Rowan Walrath Source Type: news

Pfizer joins Beam Therapeutics to develop rare disease therapies
Beam will receive $300 million in upfront payment and lead research activities till three new therapy targets are selected for development from outside the firm's existing programs, the companies said on Monday. (Source: The Economic Times Healthcare and Biotech News)
Source: The Economic Times Healthcare and Biotech News - January 10, 2022 Category: Pharmaceuticals Source Type: news

Gene Therapies Could Treat Rare Diseases — but Cost Millions Gene Therapies Could Treat Rare Diseases — but Cost Millions
A biotechnology and policy expert shines a light on the looming gene therapies soon to hit the market, and the payment strategies needed to get them to patients.Quick Take (Source: Medscape Internal Medicine Headlines)
Source: Medscape Internal Medicine Headlines - December 29, 2021 Category: Internal Medicine Tags: Internal Medicine News Source Type: news

'Unmovable monster': The drug trial that changed how biotechs approach rare disease treatments
Billy Ellsworth was 10 years old when he started traveling from his home outside Philadelphia to Columbus, Ohio, to take part in a small trial of an experimental drug to treat the rare, degenerative disease with which he was born. A decade later, his memories of that time are fleeting. He remembers knowing the drug was intended to help his muscles to stay healthy despite the effects of Duchenne muscular dystrophy. He remembers being nervous about his first biopsy, having never undergone surgery… (Source: bizjournals.com Health Care News Headlines)
Source: bizjournals.com Health Care News Headlines - December 26, 2021 Category: Health Management Authors: Don Seiffert Source Type: news

'Unmovable monster': The drug trial that changed how biotechs approach rare disease treatments
Billy Ellsworth was 10 years old when he started flying from his home outside Philadelphia to Columbus, Ohio to take part in a small trial of an experimental drug to treat the rare, degenerative disease with which he was born. A decade later, his memories of that time are fleeting. He remembers knowing the drug was intended to help his muscles to stay healthy despite the effects of Duchenne muscular dystrophy. He remembers being nervous about his first biopsy, having never undergone surgery before.… (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - December 23, 2021 Category: American Health Authors: Don Seiffert Source Type: news

TARPEYO & lt;sup & gt;TM & lt;/sup & gt; (budesonide) delayed release capsules, FDA Approved to Reduce Proteinuria in IgA Nephropathy, Available Exclusively at Biologics by McKesson
CARY, N.C., Dec. 22, 2021 —Biologics by McKesson, an independent specialty pharmacy specializing in oncology and rare disease areas, was selected by Calliditas Therapeutics as the exclusive specialty pharmacy provider for TARPEYOTM (budesonide) delayed release capsules to reduce proteinuria in adults... (Source: McKesson News)
Source: McKesson News - December 22, 2021 Category: Information Technology Source Type: news

New Report: Nearly 800 new medicines in development to treat rare diseases
Today, PhRMA released anew report detailing orphan drug development in the U.S. and the potential to meet the significant unmet medical need for patients. Rare diseases and conditions individually affect small patient populations, but collectively they impact approximately 400 million patients around the world. America ’s biopharmaceutical research companies have spent decades working to tackle these extremely challenging diseases, of which researchers have identified approximately 8,000. In the United States alone, roughly 30 million Americans are living with a rare disease. (Source: The Catalyst)
Source: The Catalyst - December 16, 2021 Category: Pharmaceuticals Tags: Medicines in Development Research and Development Rare Diseases Source Type: news

HemoShear Therapeutics Appoints Patrick Horn, MD, PhD, Chief Medical Officer
Industry Veteran Will Lead HST5040 and Future Rare Disease Clinical Programs CHARLOTTESVILLE, Va., Dec. 14, 2021 -- (Healthcare Sales & Marketing Network) -- HemoShear Therapeutics, Inc., a privately held biotechnology company developing drugs for rare... Biopharmaceuticals, Personnel HemoShear Therapeutics, REVEAL-Tx (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - December 14, 2021 Category: Pharmaceuticals Source Type: news

Nike's head of video games embraces new title: 'rare disease dad'
At three months, Tommy Pham's son began showing symptoms that puzzled the family and doctors alike. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - December 13, 2021 Category: Pharmaceuticals Authors: Elizabeth Hayes Source Type: news

NIDCR's Winter 2021 E-Newsletter
Having trouble viewing this email? View it as a Web page. NIDCR's Winter 2021 E-Newsletter In this issue: NIDCR News Funding Opportunities & Related Notices NIH/HHS News Subscribe to NIDCR News Science Advances   NIDCR News NIDCR Major Announcement: Save the Date! Mark your calendar for Tuesday, December 21, at 1pm for a major webcast announcement from NIDCR. Look for log-in instructions, coming in a separate NIDCR email next week. Jennifer Webster-Cyriaque Joins NIDCR as Deputy Director NIDCR welcomed Jennifer Webster-...
Source: NIDCR Science News - December 8, 2021 Category: Dentistry Source Type: news

BESREMi (ropeginterferon alfa-2b-njft), FDA Approved for Treatment of Polycythemia Vera, Available at Biologics by McKesson
CARY, N.C., Dec. 6, 2021 —Biologics by McKesson, an independent specialty pharmacy specializing in oncology and rare disease areas, was selected by PharmaEssentia USA Corporation as a specialty pharmacy provider for BESREMi® (ropeginterferon alfa-2b-njft) for the treatment of adults with polycythemia... (Source: McKesson News)
Source: McKesson News - December 6, 2021 Category: Information Technology Source Type: news

Newly Approved LIVTENCITY (maribavir) Now Available at Biologics by McKesson
CARY, N.C., Dec. 3, 2021 —Biologics by McKesson, an independent specialty pharmacy specializing in oncology and rare disease areas, was selected by Takeda Pharmaceuticals as a specialty pharmacy provider for LIVTENCITYTM (maribavir), indicated for the treatment of adults and pediatric patients (12 ye... (Source: McKesson News)
Source: McKesson News - December 3, 2021 Category: Information Technology Source Type: news

Scientists raise concerns over UK baby genome sequencing plan
Exclusive: experts say scheme seems designed to create valuable dataset rather than improve screeningScientists have raised concerns about a proposed overhaul of newborn screening that could lead to the UK becoming the first country to offer whole-genome sequencing for every baby.Speaking before the publication of plans for an NHS pilot study in which up to 200,000 babies ’ genomes will be sequenced and analysed, scientists suggested the initiative appeared designed to create a valuable health dataset rather than an effective method of improving the diagnosis of rare diseases.Continue reading... (Source: Guardian Unlimited Science)
Source: Guardian Unlimited Science - December 2, 2021 Category: Science Authors: Hannah Devlin Science correspondent Tags: Genetics NHS Health Health policy Biology Science Society Politics UK news Source Type: news

Doctors Treat Girl's Genetic Disorder with Repurposed Drug
In just 16 months, physicians went from identifying a novel rare disease in three-year-old Marley to successfully treating her with a drug previously used to treat African sleeping sickness and... (Source: The Scientist)
Source: The Scientist - December 1, 2021 Category: Science Tags: Notebook Magazine Issue Source Type: news