ALS Association

Mitsubishi Tanabe Pharma America Receives U.S. Food and Drug Administration Orphan Drug Exclusivity for RADICAVA ORS® (edaravone)

Retrieved on: 
Monday, April 8, 2024
MTPA, Orphan drug, ALS, RWE, ODE, Patient, ALS Association, Edaravone, Safety, HCP, FDA, Health, Food, Pharmaceutical industry

JERSEY CITY, N.J., April 8, 2024 /PRNewswire/ -- Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced that the U.S. Food and Drug Administration (FDA) has recognized seven years of Orphan Drug Exclusivity (ODE) for RADICAVA ORS® (edaravone) based on their assessment that the oral form of edaravone constitutes a major contribution to patient care for people living with amyotrophic lateral sclerosis (ALS). The FDA's Orphan Drug program is designed to support the development of drugs that treat rare diseases which affect less than 200,000 people in the U.S. Previously, the FDA granted RADICAVA ORS Priority Review and Fast Track designations.

Key Points: 
  • The FDA's Orphan Drug program is designed to support the development of drugs that treat rare diseases which affect less than 200,000 people in the U.S.
  • Previously, the FDA granted RADICAVA ORS Priority Review and Fast Track designations.
  • The FDA recognized ODE for RADICAVA ORS because it provides a clinically superior option for patients due to its oral suspension route of administration that can help reduce the burden patients face with intravenous (IV) administration of previously approved RADICAVA® (edaravone).
  • "We thank Mitsubishi Tanabe Pharma America for continuing their efforts to help provide treatments to people living with ALS."

The ALS Association Announces Inaugural 'ALS Nexus' Conference

Retrieved on: 
Friday, March 22, 2024
Nexus, Research, Conference, Care, ALS, Caregiver, Convention center, Ice Bucket Challenge, ALS Association, Hope, Movement

 ARLINGTON, Va., March 22, 2024 /PRNewswire/ -- The ALS Association, the leading national non-profit organization dedicated to fighting ALS on every front, is excited to announce its inaugural ALS Nexus Conference, taking place July 14-17, 2024 at The Gaylord Texan Resort & Conference Center in Grapevine, Texas.

Key Points: 
  • First-of-Its-Kind Gathering Will Convene People Living with ALS, Along with Leaders in ALS Research, Advocacy, and Care
    ARLINGTON, Va., March 22, 2024 /PRNewswire/ -- The ALS Association, the leading national non-profit organization dedicated to fighting ALS on every front, is excited to announce its inaugural ALS Nexus Conference , taking place July 14-17, 2024 at The Gaylord Texan Resort & Conference Center in Grapevine, Texas.
  • First-of-Its-Kind Gathering Will Convene People Living with ALS, Along with Leaders in ALS Research, Advocacy, and Care
    The ALS Association is committed to making ALS a livable disease for everyone, everywhere, until there's a cure.
  • "ALS Nexus represents a significant step forward in our efforts to transform the experience of living with ALS," stated Nicole Eck, chief strategy officer.
  • Don't miss your chance to be part of the movement changing the future of ALS, register now for the inaugural ALS Nexus Conference .

myTomorrows Partners with The ALS Association to Support ALS Patients in Accessing Clinical Trials

Retrieved on: 
Thursday, March 14, 2024
Diagnosis, ALS, Brain, Face, Disease, Partnership, Patient, ALS Association, Clinical trial, Spinal cord, Neuromuscular disease, Physician, Neurodegenerative disease, Pharmaceutical industry

NEW YORK, March 14, 2024 /PRNewswire/ -- myTomorrows today announced a new partnership with the ALS Association, the U.S.-based nonprofit organization fighting ALS on every front.

Key Points: 
  • Through the partnership, the ALS Association will tap into myTomorrows' vast database of ongoing clinical trials and leverage its patient navigation service to ensure that treating physicians and those living with ALS are equipped with up-to-date information about appropriate pre-approval treatment options and ultimately guided towards relevant clinical trials.
  • The ALS Association serves more than 20,000 people living with ALS in the United States, and its partnership with myTomorrows aims to make it easier for patients and their families to find and participate in clinical trials.
  • myTomorrows explores each patient's case individually, helping ALS patients and their loved ones identify and understand all of the available clinical trial options in a well-rounded manner, guided by expert patient navigators who demystify the complexities of clinical trials and support patients and their families throughout the process.
  • Partnering with leading patient advocacy groups such as the ALS Association will help an even larger cohort of ALS patients and their treating physicians discover clinical trials, thereby reducing the barriers they may face in accessing their treatment options."

Coave Therapeutics Receives Grant from the ALS Association to Advance its CTx-TFEB Program as a Potential Treatment for All Forms of ALS

Retrieved on: 
Thursday, February 29, 2024
ALS Association, Senior, ALS, Research, Alzheimer's disease, TFEB, Wang, Neuron, ALP, Hope, Trehalose, Neurodegenerative disease, Protein, Patient, Spinal cord, Gene, Disease, CSO, Diagnosis, Autophagy, AAV, Amyotrophy, Brain

The grant, which will support the development of Coave’s CTx-TFEB program through to preclinical proof-of-concept, has been made through the Association’s Lawrence and Isabel Barnett Drug Development Program .

Key Points: 
  • The grant, which will support the development of Coave’s CTx-TFEB program through to preclinical proof-of-concept, has been made through the Association’s Lawrence and Isabel Barnett Drug Development Program .
  • ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord.
  • Over the course of the disease, people lose the ability to move, to speak, and eventually, to breathe.
  • With this funding and invaluable support from the ALS Association, we are poised to move our CTx-TFEB program forward.

ORYZON Reports Financial Results and Corporate Update for Quarter Ended December 31, 2023

Retrieved on: 
Monday, February 26, 2024
Borderline personality disorder, HOPE, HDAC6, National Cancer Institute, EVOLUTION, CMT, Agitation, Data analysis, Convertible bond, ICIS, ALS, SCLC, Azacitidine, Duration, ICI, IIS, Sciatic nerve, Deficit spending, Paclitaxel, DSM-IV codes, Development, Genomics, ALS Association, FMS, LSD1, AML, Clinical Global Impression, MSKCC, Conference, Aggression, Safety, Biomarker, CNS, Venetoclax, CRADA, OHSU, MRD, Clinical trial, BPD, Measure (mathematics), IND, FCCC, Dor, Human, Innovation, EMA, Kabuki syndrome, NET, NCI, Aes, Glomus tumor, FDA, GST, Food, Platinum, Schizophrenia, Phase II, Charcot–Marie–Tooth disease, Medicine, Patient, BEST, Disease, Pharmaceutical industry

Dr Carlos Buesa, Oryzon’s Chief Executive Officer, said: “Oryzon continued with a strong path in its clinical programs in the fourth quarter.

Key Points: 
  • Dr Carlos Buesa, Oryzon’s Chief Executive Officer, said: “Oryzon continued with a strong path in its clinical programs in the fourth quarter.
  • Research and development (R&D) expenses were $3.9 and $16.6 million for the quarter and twelve months ended December 31, 2023, respectively, compared to $5.0 and $18.1 million for the quarter and twelve months ended December 31, 2022.
  • General and administrative expenses were $1.2 and $4.2 million for the quarter and twelve months ended December 31, 2023, respectively, compared to $1.2 and $4.8 million for the quarter and twelve months ended December 31, 2022.
  • Net losses were $1.4 and $5.0 million for the quarter and twelve months ended December 31, 2023, respectively, compared to $1.6 and $5.9 million for the quarter and twelve months ended December 31, 2022.

"LUKi & The Lights" Animated Short Film Illuminates ALS Diagnosis for Young Audiences

Retrieved on: 
Friday, March 8, 2024
Florida Film Festival, ALS Association, ALS, Doctor of Philosophy, Friends, Culture, Language, Parent, Hope, Film festival, Terminal illness, Luki, Diagnosis, LCSW, Anjo, Animation, Light, Entertainment

ARLINGTON, Va., March 7, 2024 /PRNewswire/ -- "LUKi & The Lights," an animated short film aimed at helping young audiences understand and cope with the complexities of ALS, will make its public premiere at Cinema Village in New York City on March 22. Created by Sascha Groen and her husband, Anjo Snijders, who was diagnosed with ALS in 2017 at the age of 35, "LUKi & The Lights" follows the journey of LUKi, a charming robot who navigates life after being diagnosed with ALS, an always fatal neurodegenerative disease that does not have a cure. The film leverages the power of animation to tell the story of LUKi and his friends, which makes it easy for children in any culture to understand. Through the evocative power of animation, the film transcends language barriers, allowing children worldwide to grasp the devastating nature of ALS.

Key Points: 
  • Through the evocative power of animation, the film transcends language barriers, allowing children worldwide to grasp the devastating nature of ALS.
  • He and his wife Sascha realized the need for resources to explain ALS to their own children after his diagnosis.
  • "We have already seen the tremendous reception the film has received both within the film and ALS communities.
  • During this time, audiences of all ages will have the opportunity to experience the heartfelt story of LUKi and his journey with ALS.

Hugh Hoffman's Historic Gift to ALS Association Will Elevate Fight Against ALS

Retrieved on: 
Monday, February 12, 2024
ALS Association, ALS, Research, University, Hope, History, Death, Family, Yale University, Pharmaceutical industry

ARLINGTON, Va., Feb. 12, 2024 /PRNewswire/ -- The ALS Association proudly announces the largest single gift in its history from the estate of the late philanthropist Hugh Hoffman. The $58 million gift will profoundly impact the lives of people living with ALS and their loved ones, advancing the ALS Association's goal of making ALS livable until there is a cure.

Key Points: 
  • Hoffman wanted his gift to ensure that families in the future will not have to lose their loved ones to ALS.
  • Hoffman's gift is specifically designated to advancing promising ALS therapies, developing new or strengthening existing ALS clinics, and enhancing access to ALS care for people living with ALS.
  • "We are deeply grateful to Hugh Hoffman and his family," said ALS Association President and CEO Calaneet Balas.
  • The ALS Association used these gifts to significantly accelerate the fight against ALS, including funding the discovery of new ALS treatments, expanding ALS care, and creating several global research projects.

Myrobalan Therapeutics Receives a Grant from the ALS Association to Advance its CSF1R Inhibitor Program

Retrieved on: 
Wednesday, February 7, 2024
Oncology, Health, Other Health, Clinical Trials, Pharmaceutical, Biotechnology, Myrobalan, Life expectancy, Senior, ALS, Multiple sclerosis, Clinical trial, Research, IND, Patient, Diagnosis, CNS, Brain, Therapy, Drug development, Brain cell, ALS Association, Spinal cord, Pharmaceutical industry

ALS is a relentlessly progressive fatal neurodegenerative disease affecting the motor neurons in the brain and spinal cord.

Key Points: 
  • ALS is a relentlessly progressive fatal neurodegenerative disease affecting the motor neurons in the brain and spinal cord.
  • Unlike other CSF1R candidates, Myrobalan’s CSF1R inhibitor program was specifically designed for application in CNS diseases with compelling advantages across several preclinical measures, including potency, selectivity, and CNS distribution.
  • In addition to ALS, Myrobalan plans to develop its CSF1R inhibitor in additional neurodegenerative indications such as Multiple Sclerosis and Alzheimer’s Disease.
  • Ultimately, our goal is to make a CSF1R inhibitor therapy accessible to all patients grappling with ALS.”

ALS Association Greater Philadelphia Chapter is Now ALS United Mid-Atlantic

Retrieved on: 
Tuesday, February 6, 2024
ALS Association, Growth, Knowledge, Celebration, Sodium phenylbutyrate/ursodoxicoltaurine, Research, MD, Nursing, FDA, Food, Disease, Delaware Valley, Health, Social, Doctor of Philosophy, Innovation, ALS

AMBLER, Pa., Feb. 6, 2024 /PRNewswire/ -- The ALS Association Greater Philadelphia Chapter has completed rebranding to become ALS United Mid-Atlantic.

Key Points: 
  • AMBLER, Pa., Feb. 6, 2024 /PRNewswire/ -- The ALS Association Greater Philadelphia Chapter has completed rebranding to become ALS United Mid-Atlantic.
  • This transition comes after the Greater Philadelphia Chapter joined 14 other Chapters in legally separating from The ALS Association on September 1, 2023.
  • These chapters represent approximately half of the ALS patient population and Certified Treatment Centers and ALS Clinics in the nation.
  • Videos highlighting these awards, ALS United Mid-Atlantic Treatment Centers, and a Call to Action to support the ALS Mission are available at https://www.youtube.com/@ALSMidAtlantic
    Over half of the budget of ALS United Mid-Atlantic is devoted to direct patient care.

Quorum Announces Next Generation for Grassroots Advocacy

Retrieved on: 
Monday, January 29, 2024
Noise, Pulse, Policy, ALS Association, AI, Government, System, The, Organization, ALS

WASHINGTON, Jan. 29, 2024 /PRNewswire/ -- Quorum, a market leader in public affairs software, launches the next generation of grassroots advocacy software with major new updates to Quorum Grassroots. With intuitive workflows and AI features to provide a modern advocate experience and drive greater engagement, public affairs professionals will be able to greater amplify the issues they care about.

Key Points: 
  • WASHINGTON, Jan. 29, 2024 /PRNewswire/ -- Quorum , a market leader in public affairs software, launches the next generation of grassroots advocacy software with major new updates to Quorum Grassroots.
  • Quorum Grassroots — and the full Quorum suite — empowers its users with a comprehensive grassroots and government affairs platform to maximize every interaction between organizations and those who influence policy.
  • This launch marks the integration of Phone2Action's cutting-edge grassroots mobilization and digital advocacy technology into the Quorum platform.
  • For more information on how Quorum is powering grassroots advocacy, visit www.quorum.us .