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☆ FDA advisors reject Biogen's ALS drug for rare and aggressive form of the disease
▼ + stars: | 2023-03-22 | by ( Annika Kim Constantino | ) www.cnbc.com time to read: +3 min

The Food and Drug Administration's independent panel of advisors on Wednesday declined to endorse accelerated approval of Biogen's investigational ALS drug for a rare and aggressive form of the disease. The drug tofersen was developed to treat a rare genetic form of amyotrophic lateral sclerosis, or ALS. That number is even smaller in the U.S., with roughly 330 people affected by the SOD1 mutation. The median survival time from diagnosis with the rare form of ALS to death is 2.7 years, according to the company. The SOD1 mutation is associated with 20% of cases that occur within families.

☆ FDA advisors vote against effectiveness of Biogen's ALS drug for rare and aggressive form of the disease
▼ + stars: | 2023-03-22 | by ( Annika Kim Constantino | ) www.cnbc.com time to read: +3 min

The Food and Drug Administration's independent panel of advisors on Wednesday voted against the effectiveness of Biogen's investigational ALS drug for a rare and aggressive form of the disease. The drug tofersen was developed to treat a rare genetic form of amyotrophic lateral sclerosis, or ALS. That number is even smaller in the U.S., with roughly 330 people affected by the SOD1 mutation. The median survival time from diagnosis with the rare form of ALS to death is 2.7 years, according to the company. The SOD1 mutation is associated with 20% of cases that occur within families.

☆ FDA staff says safety issues with Biogen's ALS drug to not prevent approval
▼ + stars: | 2023-03-21 | by ( Bhanvi Satija | ) www.reuters.com time to read: +2 min

March 20 (Reuters) - The U.S. health regulator's staff said on Monday safety issues with Biogen Inc's (BIIB.O) drug to treat an ultra-rare form of amyotrophic lateral sclerosis or ALS, also known as Lou Gehrig's disease, should not prevent its accelerated approval. The drug, tofersen, failed to meet its main goal of reducing symptoms but reduced protein neurofilament levels during a late-stage study. The risks related to Biogen's drug, such as spinal cord inflammation and optic nerve swelling, observed in trial patients "are acceptable to the patient population", the staff said. They added that a majority of these risks may be related to the way in which the drug is administered, rather than the drug itself. The company is seeking approval for the drug in ALS patients with mutation in a gene known as superoxide dismutase 1.

☆ US FDA staff flags no new safety concerns for Biogen's ALS drug
▼ + stars: | 2023-03-20 | by ( ) www.reuters.com time to read: +1 min

March 20 (Reuters) - Staff reviewers of U.S. drug regulator on Monday did not raise any new safety concerns about Biogen Inc's (BIIB.O) experimental drug for treating a rare type of amyotrophic lateral sclerosis, also known as Lou Gehrig's disease. The risks related to Biogen's drug, tofersen, "are acceptable to the patient population" and would not "preclude approval", staff reviewers said in briefing documents published on the agency's website. The agency has asked advisers to weigh in on approving the use of the drug based on available data, which shows it reduces a key protein related to the disease, and not disease symptoms. In October 2021, tofersen failed to meet the main goal of a late-stage study, but the company said trends of reduced disease progression were observed. Reporting by Raghav Mahobe and Bhanvi Satija in Bengaluru; Editing by Krishna Chandra Eluri and Shinjini GanguliOur Standards: The Thomson Reuters Trust Principles.

☆ FDA staff says Biogen's ALS drug may have a 'clinical benefit' on a rare form of the disease
▼ + stars: | 2023-03-20 | by ( Annika Kim Constantino | ) www.cnbc.com time to read: +6 min

U.S. Food and Drug Administration staff on Monday said Biogen's investigational ALS drug may have a "clinical benefit" on a rare and aggressive form of the disease, despite failing a broader late-stage clinical trial last year. A separate exploratory analysis of the drug suggests "a clinical benefit with a longer duration of treatment," the staff wrote. It also found that tofersen reduced levels of a protein called neurofilament light (NfL,) which is "correlated with disease severity, disease progression rate, and survival in patients with ALS," according to the FDA staff. The first is to move forward with recommending accelerated approval based on tofersen's reduction of NfL in ALS patients. "SOD1-linked ALS is a particularly rare and aggressive form of an already rare and devastating disease," said Dr. Neil Thakur, The ALS Association's chief mission officer.

☆ Thousands sign petition to save 'sacred' Japan stadium where Ruth once played
▼ + stars: | 2023-01-17 | by ( Elaine Lies | ) www.reuters.com time to read: +2 min

TOKYO, Jan 17 (Reuters) - Thousands of baseball fans have signed a petition to save an iconic Tokyo stadium nearly a century old where Babe Ruth once played and which inspired best-selling author Haruki Murakami to first pick up a pen. "The citizens of Tokyo are going to regret it," said Robert Whiting, who has written books on Japanese baseball and who over the weekend started an online petition to save the stadium, which "reeks of history." "They're going to lose a really beautiful, quiet, relaxing spot and a great place to watch a baseball game," he told Reuters. Babe Ruth and Lou Gehrig played there in 1934 as part of a Japanese tour, making the stadium only one of a handful remaining where Ruth played. By noon on Tuesday, Whiting's petition, addressed to Tokyo governor Yuriko Koike and several others, had almost 10,000 signatures.

☆ A new test for autism hopes to help doctors diagnose before symptoms show
▼ + stars: | 2023-01-05 | by ( Evan Bush | Evan Bush Is A Science Reporter For Nbc News. He Can Be Reached At Evan.Bush Nbcuni.Com. | ) www.nbcnews.com time to read: +8 min

Researchers have discovered myriad risk factors associated with autism, including infections during pregnancy, air pollution and maternal stress. “We can detect the clear rhythm of autism with just about one centimeter of hair,” Arora said. Autism diagnosis timingArora and his team hope their technology could help young children, even newborns, receive early interventions for autism sooner than they can now. “We want to enable early intervention.”There is not yet a biological test for autism spectrum disorder. Perhaps eventually, that information could open up new pathways for the development of drugs or therapies for autism, he said.

☆ Fanatics is divesting its 60% stake in NFT company Candy Digital
▼ + stars: | 2023-01-04 | by ( Ian Thomas | ) www.cnbc.com time to read: +3 min

Michael Rubin's sports platform company Fanatics is divesting its 60% stake in NFT company Candy Digital, according to an internal email obtained by CNBC. Candy Digital was founded in June 2021 in the middle of the sports NFT boom, competing with companies like Dapper Labs in the digital sports collectible space. It also released digital collectibles with Netflix 's Stranger Things, WWE , and several Nascar teams. Dapper Labs, the company behind NBA Top Shot and NFL All Day digital trading platforms that ranked No. "Over the past year, it has become clear that NFTs are unlikely to be sustainable or profitable as a standalone business," Rubin wrote.

☆ Final farewell: From Sidney Poitier to Pope Emeritus Benedict a look back at the influential figures who died in 2022
▼ + stars: | 2023-01-01 | by ( Associated Press | ) www.nbcnews.com time to read: +55 min

Other world leaders who died in 2022 include former Soviet leader Mikhail Gorbachev, who died in August. The final days of 2022 saw the loss of some exceptionally notable figures, including Pope Emeritus Benedict XVI. Here is a roll call of some influential figures who died in 2022 (cause of death cited for younger people, if available):___JANUARY___Dan Reeves, 77. A Cuban-born artist whose radiant color palette and geometric paintings were overlooked for decades before the art world took notice. A prolific character actor best known for playing villains and tough guys in “The Manchurian Candidate,” “Ocean’s Eleven” and other films.

☆ Police officers to receive Congressional Gold Medals for Jan. 6
▼ + stars: | 2022-12-06 | by ( Associated Press | ) www.nbcnews.com time to read: +5 min

To recognize the hundreds of officers who were at the Capitol on Jan. 6, the medals will be placed in four locations — at U.S. Capitol Police headquarters, the Metropolitan Police Department, the Capitol and the Smithsonian Institution. Awarding the medals will be among House Speaker Nancy Pelosi’s last ceremonial acts as she prepares to step down from leadership. Officers suffered physical wounds, including brain injuries and other lifelong effects, and many struggled to work afterward because they were so traumatized. Two police officers died by suicide in the days that immediately followed, and a third officer, Capitol Police Officer Brian Sicknick, collapsed and later died after one of the rioters sprayed him with a chemical. The Congressional Gold Medal, the highest honor Congress can bestow, has been handed out by the legislative branch since 1776.

☆ FDA considering shift toward individual risk-based assessments for blood donors
▼ + stars: | 2022-11-30 | by ( Carma Hassan | ) edition.cnn.com time to read: +4 min

CNN —The US Food and Drug Administration is considering shifting its blood donation policy away from blanket assessments toward questionnaires that focus more on individual risk, it said Wednesday. “The FDA remains committed to gathering the scientific data related to alternative donor deferral policies that maintain a high level of blood safety,” the statement says. Those who have not would be allowed to donate blood, an FDA official told the Journal. The current policy requires men who have sex with men to wait three months after sexual contact with other men before they can donate blood. Other men who have sex with men are still required to refrain from sex for three months before donating in the UK.

☆ Singer Roberta Flack Has ALS, Finds It ‘Impossible to Sing’
▼ + stars: | 2022-11-15 | by ( Ginger Adams Otis | ) www.wsj.com time to read: 1 min

Roberta Flack won back-to-back Grammy awards for record of the year in 1973 and 1974. Grammy award-winning artist Roberta Flack has ALS, commonly known as Lou Gehrig ‘s disease, and can no longer sing, her representative said Monday. Ms. Flack, 85, had hoped to attend Thursday’s premiere of “Roberta,” a feature-length documentary about her that’s being screened at DOC NYC, an annual documentary film festival in New York.

☆ Roberta Flack has ALS, now ‘impossible to sing,’ rep says
▼ + stars: | 2022-11-14 | by ( Associated Press | ) www.nbcnews.com time to read: +3 min

A representative for Roberta Flack announced Monday that the Grammy-winning musician has ALS, commonly known as Lou Gehrig’s disease, and can no longer sing. The progressive disease “has made it impossible to sing and not easy to speak,” Flack’s manager Suzanne Koga said in a release. Flack also plans to publish a children’s book co-written with Tonya Bolden, “The Green Piano: How Little Me Found Music,” that month. Flack had a stroke in 2016 and spoke to The Associated Press a little over two years later about returning to performing. “I could sing any number of songs that I’ve recorded through the years, easily, I could sing them, but I’m going to pick those songs that move me,” Flack said.

☆ Roberta Flack can no longer sing after ALS diagnosis
▼ + stars: | 2022-11-14 | by ( Story Reuters | ) edition.cnn.com time to read: +1 min

Grammy-winning musician Roberta Flack, whose hits include “Killing Me Softly with His Song,” has been diagnosed with ALS, also known as Lou Gehrig’s disease, and can no longer sing, her representatives said on Monday. Amyotrophic lateral sclerosis, a progressive disease, “has made it impossible to sing and not easy to speak,” a statement from her publicists said. “But it will take a lot more than ALS to silence this icon.”A documentary about Flack’s life is set to debut at a film festival in New York next week. Flack, 85, also is releasing a children’s book in January. ALS breaks down nerve cells in the brain and spinal cord that make muscles work, leading to progressive paralysis and death.

☆ Sam Shields: Super Bowl champion regrets playing in NFL; says his head is 'all mushed together' with concussions
▼ + stars: | 2022-11-03 | by ( Ben Morse | ) edition.cnn.com time to read: +7 min

CNN —Former Green Bay Packers cornerback Sam Shields has said he regrets playing in the NFL, pointing to the effects concussions and head injuries had on his career and his later life. The spotters “serve as another set of eyes, watching for possible injuries at every NFL game,” according to NFL Football Operations. Concussions and their prevention have become an important issue in recent years due to their connection to brain disease later in life. The neurodegenerative brain disease can be found in individuals who have been exposed to repeated head trauma. The researchers hypothesized about a relationship between head trauma and ALS because of a similar link detected between football and the neurodegenerative disease CTE.

☆ Startup Parrots is using 5G to develop a bird-shaped, AI-powered machine that helps people with disabilities communicate
▼ + stars: | 2022-10-27 | by ( Karen Fischer | ) www.businessinsider.com time to read: +6 min

Polly, a hardware and software service, helps people with disabilities live more independently. Parrots' founder is in talks with the VA to give Polly to veterans with neurological disorders. This article is part of "How 5G Is Changing Everything," a series about transformational 5G tech across industries. Hojah acknowledged that Parrots could have taken flight without 5G but said the timeline would have been much slower: "5G is expanding our growth rapidly." In 2019, Parrots won Verizon's Built on 5G Challenge, and Hojah got mentorship and technological assistance from the wireless-telecommunications giant.

☆ Fetterman Oz debate for Pennsylvania Senate seat sends message to stroke survivors
▼ + stars: | 2022-10-25 | by ( Judith Hannah Weiss | Judith Hannah Weiss Is A Freelance Writer Whose Work Has Appeared In The Iowa Review | The Rumpus | ) www.nbcnews.com time to read: +7 min

John Fetterman suffered days before winning the Democratic Senate nomination in May. The stroke added a new wrinkle to a race that could determine which party takes control of the 50-50 U.S. Senate. The Oz campaign is clearly hoping that perception will hurt Fetterman. In one egregious example, Oz issued a list of “concessions” for their debate Tuesday night that many see as mocking. Benjamin Abella, a professor of emergency medicine at the University of Pennsylvania, criticized the Oz campaign for shaming a stroke survivor.

☆ Why a $158,000 Drug With Unclear Benefits Hurts Whole Health System
▼ + stars: | 2022-10-10 | by ( David Wainer | ) www.wsj.com time to read: 1 min

Like many patients suffering from amyotrophic lateral sclerosis, or ALS—also known as Lou Gehrig’s disease—Layne Oliff didn’t have any time to waste. Even before the drug Relyvrio was approved late last month by the Food and Drug Administration, he has had his own do-it-yourself method: he gets sodium phenylbutyrate in liquid form from a New Jersey pharmacy and taurursodiol online from Amazon. That costs him over $7,000 a year, but he says it has been well worth it because he feels the combination has helped stabilize a disease that often causes death within a few years.

☆ A controversial ALS drug potentially worth $1 billion is on the cusp of FDA approval. It could change Amylyx's fortunes and be a much-needed treatment for the rare disease.
▼ + stars: | 2022-09-26 | by ( Yeji Jesse Lee | ) www.businessinsider.com time to read: +7 min

The FDA may soon approve a new treatment for ALS, also known as Lou Gehrig's disease. If approved, Amylyx Pharmaceuticals's drug, AMX0035, will be a new treatment option for patients. A new drug for amyotrophic lateral sclerosis, more commonly known as ALS or Lou Gehrig's disease, is on the cusp of approval from the US Food and Drug Administration. The association said that treatment has the support of the larger ALS community and urged the FDA to "swiftly approve" AMX0035. By comparison, there are around 30,000 ALS patients in the US, meaning reimbursing AMX0035 would pose a much lighter burden on the CMS.

☆ Biogen says ALS drug shows clinical benefit in new data analysis
▼ + stars: | 2022-09-21 | by ( Mrinalika Roy | ) www.reuters.com time to read: +2 min

The Biogen drug, tofersen, is currently under priority review by the U.S. Food and Drug Administration with an approval decision expected by Jan. 25. Biogen last October had said the drug missed the main goal of the late-stage study, failing to show statistically significant improvement in the functional status of patients with fast-progressing ALS at six months. Biogen is seeking approval of tofersen for ALS patients with mutations in a specific gene that leads to accumulation of toxic levels of a protein called SOD1. Most side effects in both the late-stage study and the follow-on portion were mild to moderate, including headache and back pain, the company said. Nearly 7% of patients who received tofersen experienced serious neurological events, including spinal cord inflammation and swelling of the optic nerve.

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